Synopulse Catalyst
Rare Disease Catalyst Report: Non-Oncology Pipeline & Milestones
October 2026
Monthly overview of non-oncology rare-disease research, clinical data, regulatory and policy milestones, deals and funding, and the catalysts due next.
What we’re watching this month
Shionogi Paid $2bn for an NPC Drug in October, and the FDA Rules on Beren's Competing Therapy on 17 November
Rare-disease deals carried $3.4bn in disclosed upfront and voucher value, led by Shionogi's purchase of IntraBio, as IgA nephropathy gained its first complement inhibitor. November brings six FDA action dates, from Beren's adrabetadex in Niemann-Pick type C on 17 November to Vertex's povetacicept on 30 November.
Shionogi's $2,000m purchase of IntraBio was the largest of October's 11 qualifying deals, and by the end of the month two rival drugs for the same disease were before the FDA. Financings of $30m or more raised $1,495m, led by Disc Medicine's $450m offering two days after its Phase 3 sweep in erythropoietic protoporphyria. Regulators approved six drugs, including AstraZeneca's Ultomiris in IgA nephropathy and INOVIO's VOCERYQ in recurrent respiratory papillomatosis.
Niemann-Pick type C could have four drugs by early 2027
AQNEURSA, which Shionogi is buying, and Zevra's Miplyffa are approved. Beren's adrabetadex reaches its FDA action date on 17 November, backed by data showing more than 10 years of preserved neurological function in infantile-onset patients. Cyclo Therapeutics, part of Rafael Holdings, filed remibetadex on 28 October although its Phase 3 missed the primary endpoint in the overall population.
IgA nephropathy added a second mechanism while four APRIL drugs reported
Ultomiris won accelerated approval on 21 October, the first C5 complement inhibitor in the disease. The next day at ASN Kidney Week, Otsuka, Vera and RemeGen presented final or two-year Phase 3 data for sibeprenlimab, atacicept and telitacicept, and Vertex's povetacicept reaches its FDA date on 30 November.
Four APRIL-blocking drugs and one complement inhibitor will compete for IgA nephropathy by the end of November.
Phase 3 wins for BMS and Disc came alongside misses for AstraZeneca and Syndax
BMS's admilparant met its IPF Phase 3 primary endpoint at 60 mg but not at 120 mg, and Syndax's axatilimab missed in Phase 2 a day later. AstraZeneca's efzimfotase missed its HICKORY primary endpoint in adults with hypophosphatasia but remains under priority review. Disc's bitopertin met every APOLLO endpoint and will be resubmitted by year end.
Where we would change our mind
If the FDA approves adrabetadex on 17 November with a label covering infantile-onset patients, AQNEURSA meets a new rival before Shionogi has closed the deal. A rejection would leave remibetadex, filed on a missed primary endpoint, as the only near-term newcomer, and Shionogi's price would look better protected.
Companies in this issue
- Aardvark Therapeutics
- Agios Pharmaceuticals
- Alaunos Therapeutics
- Alcon
- Alentis Therapeutics
- Alesta Therapeutics
- Altay Therapeutics
- Alterity Therapeutics
- AM-Pharma
- Ambrose Healthcare
- Ambry Genetics
- Amprion
- AnnJi Pharmaceutical
- Arbor Biotechnologies
- Ascendis Pharma
- AstraZeneca
- Atsena Therapeutics
- Belite Bio
- Benitec Biopharma
- Beren Therapeutics
- BioCryst Pharmaceuticals
- Biohaven
- BioMarin
- BridgeBio Pharma
- Bristol Myers Squibb
- Calico Life Sciences
- Calluna Pharma
- Capricor Therapeutics
- Castle Creek Biosciences
- Clene
- Climb Bio
- Coya Therapeutics
- CSL
- Cyclo Therapeutics
- Disc Medicine
- Dyne Therapeutics
- Egetis Therapeutics
- Endeavor BioMedicines
- Epicore Biosystems
- EpiSign
- Evogene
- Ferrer
- GC Biopharma
- Genentech
- Geneyx
- Grit Bio
- Hanmi Pharm
- Harbour BioMed
- Idorsia
- Illumina
- INmune Bio
- Innovent Biologics
- INOVIO Pharmaceuticals
- IntraBio
- Ipsen
- Italfarmaco
- JCR Pharmaceuticals
- Kinea Bio
- Korro Bio
- Kyverna Therapeutics
- Latus Bio
- Longeveron
- Marea Therapeutics
- MavriX Bio
- MECFA
- Mediar Therapeutics
- Mirum Pharmaceuticals
- MSN Laboratories
- Nacuity Pharmaceuticals
- Nanoscope Therapeutics
- Neurizon Therapeutics
- Neurotech Pharmaceuticals
- Novo Nordisk
- Nuevocor
- Octant
- OMass Therapeutics
- Omeros
- Oorja Bio
- Orvida Pharma
- Otsuka
- PacBio
- Palvella Therapeutics
- Paradigm Therapeutics
- Pelthos Therapeutics
- Pharming Group
- Pharvaris
- Prilenia Therapeutics
- Purespring Therapeutics
- Quoin Pharmaceuticals
- Rafael Holdings
- Rectify Pharmaceuticals
- Regeneron
- RemeGen
- Rezolute
- Rhythm Pharmaceuticals
- Roche
- Rocket Pharmaceuticals
- RougeTx
- RyCarma Therapeutics
- Sanofi
- Sapreme Therapeutics
- Sarepta Therapeutics
- Satellos Bioscience
- Savara
- Scholar Rock
- Setara Biosciences
- Shionogi
- Sling Therapeutics
- Solid Biosciences
- Star Therapeutics
- Stoke Therapeutics
- Syndax Pharmaceuticals
- Synfini
- Takeda
- Tanabe Pharma
- Transcripta Bio
- TychoBio
- UCB
- Ultragenyx
- Velaris Therapeutics
- Vera Therapeutics
- VeriSIM Life
- Vertex
- Vivacta Bio
- Vor Bio
- Wave Life Sciences
- XtalPi
- Zenas BioPharma
- Zevra Therapeutics
- Zhimeng Biopharma
- Zydus Lifesciences
$3,388m
disclosed upfront and voucher sale value across six deals, led by Shionogi's $2,000m for IntraBio
$1,495m
raised in seven financings of $30m or more; Disc Medicine's $450m offering was the largest
6
approvals, among them Ultomiris in IgA nephropathy, Enspryng in thyroid eye disease and VOCERYQ in RRP
6
FDA action dates in November, from adrabetadex on 17 November to povetacicept on 30 November
Moves that matter
A $2bn bet on Niemann-Pick type C, a new mechanism in IgA nephropathy, a split dose in IPF and a third FOP drug led October
1
Shionogi (TSE: 4507); IntraBioAQNEURSA (levacetylleucine), approved in the US for NPC and ataxia-telangiectasia and in the EU for NPC
$2,000m agreed on 5 October. Closing planned for November to December
| Event | Asset | Indication | Timing | Comment |
|---|---|---|---|---|
| Acquisition agreed | AQNEURSA (levacetylleucine) | Niemann-Pick type C; ataxia-telangiectasia | 5 Oct 2026 | $2,000m; no contingent payments disclosed. |
| Closing | AQNEURSA | Niemann-Pick type C | Nov to Dec 2026 | Planned window. |
| CompetitorFDA action date | Adrabetadex; Beren Therapeutics (private) | Infantile-onset NPC | 17 Nov 2026 | Extended three months after a major amendment. More than 10 years of preserved neurological function presented on 15 October. |
| CompetitorNDA submitted with a voucher request | Remibetadex; Cyclo Therapeutics, part of Rafael Holdings | Niemann-Pick type C | 28 Oct 2026 | TransportNPC missed its primary endpoint in the overall population. |
| CompetitorMarketed | Miplyffa (arimoclomol); Zevra Therapeutics | Niemann-Pick type C | US since 2024 | The other approved oral therapy. |
CI Intel Shionogi is paying $2bn to lead a market that could hold four products by early 2027. Both cyclodextrins aim at the same NPC patients as AQNEURSA and Miplyffa, and Beren's decision lands before Shionogi has closed the deal. Beren built its case on long-term neurological preservation in infantile-onset disease. Cyclo filed on a missed primary endpoint, so its review will turn on subgroup and secondary data. Our read is that AQNEURSA's second approved indication in ataxia-telangiectasia is what protects the price Shionogi paid. Watch the 17 November label. Sources: Shionogi release, 5 Oct 2026; Beren release, 28 May 2026; Beren release, 15 Oct 2026; Rafael Holdings release, 29 Oct 2026. Checked 31 October 2026. | ||||
2
AstraZeneca (AZN), AlexionUltomiris (ravulizumab), the first C5 complement inhibitor approved in IgA nephropathy
Accelerated approval on 21 October
| Event | Asset | Indication | Timing | Comment |
|---|---|---|---|---|
| FDA accelerated approval | Ultomiris (ravulizumab) | IgA nephropathy, adults | 21 Oct 2026 | UPCR reduced 43.4% at week 34, per AstraZeneca. |
| CompetitorFinal Phase 3 VISIONARY results at ASN | Voyxact (sibeprenlimab), anti-APRIL; Otsuka | IgA nephropathy | 22 Oct 2026 | Two-year eGFR stabilised. |
| CompetitorORIGIN 3 final efficacy analysis; NEJM | Trutakna (atacicept), TACI-Fc; Vera Therapeutics (VERA) | IgA nephropathy | 22 Oct 2026 | |
| CompetitorTELIGAN two-year data | Telitacicept, TACI-Fc; RemeGen, with Vor Bio outside China | IgA nephropathy | 22 Oct 2026 | eGFR slope -0.6 against -5.7 mL/min/1.73 m2 a year on placebo. |
| CompetitorFDA action date, accelerated approval | Povetacicept, BAFF and APRIL inhibitor; Vertex (VRTX) | IgA nephropathy | 30 Nov 2026 | Reviewed under a priority review voucher. |
CI Intel Complement inhibition now sits beside four drugs that block APRIL, alone or with BAFF. The B-cell class used ASN Kidney Week, the day after the Ultomiris approval, to put two-year kidney function data in front of nephrologists, which is the evidence full approval will need. Ultomiris is already sold in PNH, aHUS, gMG and NMOSD, so Alexion enters IgAN with a mechanism that targets downstream complement injury rather than the immune complexes that drive the disease. Our read is that Ultomiris will be positioned for patients with complement activation, while the APRIL drugs compete for first line. Watch Vertex's 30 November decision. Sources: AstraZeneca US release, 21 Oct 2026; Otsuka release, 22 Oct 2026; Vera release, 22 Oct 2026; TELIGAN release, 22 Oct 2026; Vertex. Checked 31 October 2026. | ||||
3
Bristol Myers Squibb (BMY)admilparant, an oral LPA1 antagonist
ALOFT-IPF met its primary endpoint at the lower dose on 11 October
| Event | Asset | Indication | Timing | Comment |
|---|---|---|---|---|
| ALOFT-IPF Phase 3 topline | Admilparant | Idiopathic pulmonary fibrosis | 11 Oct 2026 | 60 mg met the FVC primary endpoint at week 52; 120 mg did not reach significance. About 1,255 patients. |
| ALOFT-PPF Phase 3 topline | Admilparant | Progressive pulmonary fibrosis | Early 2027 | |
| CompetitorMAXPIRe Phase 2 missed on FVC | Axatilimab, anti-CSF-1R; Syndax (SNDX) | IPF | 12 Oct 2026 | |
| CompetitorWHISTLE-PF Phase 2b met primary endpoint | Taladegib (ENV-101); Endeavor BioMedicines (private) | IPF | 8 Oct 2026 | 213 patients; effect size not disclosed. |
| CompetitorWISPer Phase 2 enrolment complete | MTX-463, anti-WISP-1; Mediar Therapeutics (private) | IPF | 7 Oct 2026 | 168 patients. |
| CompetitorAURORA Phase 2 topline | CAL101; Calluna Pharma (private) | IPF | Q1 2027 | Dosing complete 13 Oct. |
| CompetitorORBIT-IPF Phase 2 started | ORJ-001; Oorja Bio (private) | IPF | 13 Oct 2026 | |
CI Intel BMS has a positive Phase 3 in IPF, but only at the lower of two doses. A win at 60 mg and a miss at 120 mg will draw regulatory questions on exposure and response, and ALOFT-PPF in early 2027 becomes the test of whether the effect is real. Syndax's miss a day later shows how hard the FVC endpoint remains for new mechanisms. Endeavor already has positive Phase 2b data, Calluna reports in the first quarter of 2027 and Mediar has finished enrolling. Our read is that BMS now leads the next wave of oral IPF drugs, and the size of the FVC benefit will decide its value. Sources: BMS release, 11 Oct 2026; Syndax release, 12 Oct 2026; Endeavor release, 8 Oct 2026; Mediar release, 7 Oct 2026; Calluna release, 13 Oct 2026. Checked 31 October 2026. | ||||
4
Regeneron (REGN)Pasatru (garetosmab), an anti-activin A antibody approved in the US for FOP
No new heterotopic ossification lesions in weeks 56 to 84 of OPTIMA
| Event | Asset | Indication | Timing | Comment |
|---|---|---|---|---|
| OPTIMA extension data at ASBMR | Pasatru (garetosmab) | Fibrodysplasia ossificans progressiva, adults | 12 Oct 2026 | Zero new HO lesions from week 56 to 84. |
| CompetitorPart 1 preliminary efficacy, Phase 2/3 | Andecaliximab; āshibio (private) | FOP | 13 Oct 2026 | 86.2% reduction in new flare-up rate. |
| CompetitorPROGRESS cohort 2 positive, ages 6 to under 12 | Atebrioz (zilurgisertib); Mirum Pharmaceuticals (MIRM) | FOP | 26 Oct 2026 | Approved for ages 12 and over in September; sNDA for younger children planned 1H 2027. |
| CompetitorMarketed | Sohonos (palovarotene); Ipsen | FOP | US since 2023 | The first approved FOP drug. |
CI Intel FOP now has three approved drugs, and the contest has moved to durability and younger children. Regeneron's extension data address durability in adults, Mirum's cohort 2 opens the path to children aged 6 to 11, and āshibio's flare-up data put a fourth mechanism into a very small disease. Our read is that the paediatric label decides share, because FOP is usually diagnosed in early childhood. Watch Mirum's sNDA in the first half of 2027. Sources: Regeneron release, 12 Oct 2026; āshibio release, 13 Oct 2026; Mirum release, 26 Oct 2026. Checked 31 October 2026. | ||||
News by stage
- Discovery
- Clinic entry
- Readouts
- Regulatory
- Designations
- Access
- Deals
- Funding
Three companies nominated preclinical candidates, among them XtalPi's first IgA nephropathy siRNA and three ALS gene editors from Arbor, and 11 more reported preclinical results or proof of concept
Company announcements only. Each row carries a standard milestone: platform or partnership, candidate nominated, preclinical results, proof of concept or IND-enabling work complete; biomarkers move from validation results to clinical testing and clinical use.
| Date | Company or institution | Event | Asset | Indication | Notes |
|---|---|---|---|---|---|
| Discovery platforms and partnerships | |||||
| Italfarmaco and VeriSIM Life (both private) | AI discovery partnership signed | HDAC inhibitors, discovery stage | – | VeriSIM's BIOiSIM and AtlasGEN platforms applied to HDAC chemistry for neuromuscular and fibrotic disease. Potential value over $100m in fees, milestones and royalties. | |
| EpiSign and Geneyx (both private) | Platform integration: episignature analysis added to genomic interpretation | EpiSign METRIC episignature analysis | – | Covers more than 300 conditions. A 5-base version reads Illumina, PacBio and Nanopore methylation data, research use only. | |
| PacBio (PACB) and TychoBio (private) | Platform collaboration: long-read RNA data to train oligonucleotide design models | HiFi and Kinnex RNA sequencing | – | More than 10,000 samples linking antisense designs to transcriptome effects; siRNA to follow. No target or terms disclosed. | |
| Illumina (ILMN) | Platform release: SpliceAI2 splice-variant prediction model | Splice-variant prediction model | – | Trained on 100 times more data than SpliceAI; found 17% more disease-relevant splice variants in a Genomics England cohort. | |
| Evogene (EVGN) | Discovery programme: target indication named for AI-designed small molecule | First-in-class small molecule, discovery stage | Steroid-refractory acute graft-versus-host disease | ||
| Enters preclinical: candidate nominated | |||||
| Transcripta Bio and Synfini (both private) | Candidate nominated: lead small molecule against MSH3 | Unnamed small molecule | Huntington's disease | Reached in weeks, against an industry benchmark of 12 to 18 months, using transcriptomic AI and robotic synthesis. | |
| XtalPi (HKEX: 2228) | Candidate nominated: first siRNA preclinical candidate, nine months after programme start | Unnamed siRNA | IgA nephropathy | Subnanomolar silencing potency; a single dose held target-protein suppression for months in non-human primates. Target not disclosed. | |
| Arbor Biotechnologies (private) | Candidates nominated: three gene-editing development candidates | Three gene-editing candidates | ALS, familial and sporadic | Company says the programmes could address up to 97% of people with ALS. | |
| Preclinical results | |||||
| SOLVE FSHD; Tanabe Pharma | Preclinical results: nonclinical data at the World Muscle Society congress | SFH-4090 (MT-4090), DUX4 inhibitor | FSHD | Compound discovered by Tanabe Pharma. | |
| Quoin Pharmaceuticals (QNRX) | Preclinical results: mechanism paper and paediatric case report accepted by Dermatology and Therapy | QRX003, topical | Netherton syndrome | ||
| Stoke Therapeutics (STOK) | Preclinical results: disease-modification data at AAO 2026 | STK-002, antisense oligonucleotide | Autosomal dominant optic atrophy | ||
| Sarepta Therapeutics (SRPT) | Preclinical results: nuclear knockdown of toxic DMPK RNA at OTS 2026 | SRP-1003, siRNA | Myotonic dystrophy type 1 | ||
| Sapreme Therapeutics (private) | Preclinical results: oral presentation at OTS 2026 | SPM-2551 | Duchenne muscular dystrophy | Named as Sapreme's lead Duchenne candidate. | |
| Korro Bio (KRRO) | Preclinical results: lung and liver disease endpoints | KRRO-111, RNA editing | Alpha-1 antitrypsin deficiency | ||
| Rectify Pharmaceuticals (private) | Preclinical results: translational data in an oral session | RTY-406 | Primary sclerosing cholangitis | ||
| Purespring Therapeutics (private) | Preclinical results: new data as PS-003 advances toward the clinic | PS-003, podocyte-targeted gene therapy | X-linked Alport syndrome | ||
| Atsena Therapeutics (private) | Preclinical results: MYO7A expression and safety | ATSN-301, gene therapy | Usher syndrome type 1B | ||
| Proof of concept | |||||
| Kinea Bio (private) | Proof of concept: dual-AAV approach published in Science Translational Medicine | KNA-155, split-intein dual-AAV gene therapy | Dysferlinopathy (LGMDR2) | Split intein and myotropic capsids deliver the full-length dysferlin gene, too large for one AAV. | |
| Latus Bio (private) | Proof of concept: somatic instability model published in Nature Communications | – | Huntington's disease | Model and preclinical work support targeting somatic CAG repeat instability. | |
| Biomarkers and diagnostics | |||||
| Amprion (private) | Biomarker validation results: skin seed amplification assay in npj Parkinson's Disease | Skin-biopsy seed amplification assay | Multiple system atrophy | Separated MSA from Parkinson's specimens and agreed with CSF results. Research use now; US clinical launch planned within 12 months. | |
| Epicore Biosystems (private) and MECFA | Biomarker moves to clinical testing: wearable sweat chloride test | Chloride Tracker wearable, in development | Cystic fibrosis | Marmara University, Istanbul, leads clinical testing across MECFA's Middle East, North Africa and Central Asia members. | |
| Ambry Genetics (private) | Diagnostic moves to clinical use: GenomeNext whole genome sequencing test launched | GenomeNext whole genome sequencing test | – | Aimed at faster diagnosis of rare diseases. | |
- Discovery
- Clinic entry
- Readouts
- Regulatory
- Designations
- Access
- Deals
- Funding
Idorsia started two Phase 3 trials of oral lucerastat in Fabry disease, and Nacuity dosed the first patient in a confirmatory retinitis pigmentosa trial
Phase 2 and Phase 3 starts only.
| Date | Company or institution | Event | Asset | Indication | Notes |
|---|---|---|---|---|---|
| Phase 2 and Phase 3 starts | |||||
| Marea Therapeutics, a Lisata (LSTA) subsidiary | First patient dosed, Phase 2 | MAR002, GHR antagonist antibody | Acromegaly | About 72 patients, three doses after a 900 mg load, against placebo; primary endpoint IGF-1 reduction at 10 weeks. | |
| AnnJi Pharmaceutical | US sites opening for pivotal Phase 3 ROMA-KD | Rosolutamide (AJ201), oral | Spinal and bulbar muscular atrophy | About 200 ambulatory patients, global and placebo-controlled. FDA Fast Track; FDA and EU orphan designations. | |
| Oorja Bio (private) | ORBIT-IPF Phase 2 initiated | ORJ-001 | Idiopathic pulmonary fibrosis | Randomised, placebo-controlled, two-part study. Moves that matter #3 | |
| Paradigm Therapeutics | First patient enrolled in Phase 3 | SD-101 (Zorblesia) | Epidermolysis bullosa | ||
| RyCarma Therapeutics (private) | First patient dosed, Phase 2 | Surlorian, oral small molecule | RYR1-related myopathy | ||
| Nacuity Pharmaceuticals (private) | First patient dosed in global confirmatory Phase 3 | NPI-001 | Retinitis pigmentosa | SLO-RP Preserve trial; sites enrolling. | |
| Idorsia (SIX: IDIA) | Pivotal Phase 3 programme started; Nxera co-development deal | Lucerastat, oral | Fabry disease | FAB-KLEAR (16 adult men) and FAB-SWITCH (74 against enzyme replacement). Nxera pays CHF 7m upfront for Japan and Asia Pacific ex-China. | |
- Discovery
- Clinic entry
- Readouts
- Regulatory
- Designations
- Access
- Deals
- Funding
Disc swept every APOLLO endpoint and BMS won ALOFT-IPF at its lower dose, while AstraZeneca's efzimfotase and Syndax's axatilimab missed
Phase 2 and Phase 3 data. Earlier-phase data appear only when they support a pending approval: Beren's long-term adrabetadex data are kept for its 17 November FDA decision.
| Date | Company or institution | Event | Asset | Indication | Notes |
|---|---|---|---|---|---|
| Neuromuscular and neurology | |||||
| Capricor Therapeutics (CAPR) | HOPE-3 open-label extension data on upper-limb function | Deramiocel, cell therapy | Duchenne muscular dystrophy | Patients switched from placebo slowed upper-limb decline by 76% against their own prior year. | |
| BridgeBio Pharma (BBIO) | Cardiac and disease markers restored toward normal | BBP-418 (ribitol) | LGMD2I/R9 | FDA action date 27 November. | |
| Alterity Therapeutics (ASX: ATH) | New analyses of Phase 2 data | ATH434, iron chaperone | Multiple system atrophy | 50 mg slowed decline on 11-item UMSARS by about 52% against placebo at week 52, with CSF NfL as a covariate. | |
| Coya Therapeutics (COYA) | Enrolment complete, Phase 2/3 ALSTARS | COYA 302 | ALS | About 120 patients, two regimens against placebo; ALSFRS-R at 24 weeks, then a blinded extension. Topline early Q2 2027. | |
| Kyverna Therapeutics (KYTX) | Full one-year registrational KYSA-8 data at MSToronto | Miv-cel (KYV-101), autologous CD19 CAR-T | Stiff person syndrome | Benefit across all primary and secondary endpoints at one year; nearly all patients off chronic immunotherapy. | |
| University of Colorado Anschutz | Phase 2 CAR-T study reports lasting improvements | CD19 CAR-T | Stiff person syndrome | Academic study. | |
| Vor Bio (VOR) | Enrolment complete in Phase 3 UPSTREAM MG | Telitacicept, TACI-Fc | Generalised myasthenia gravis | Topline guided to 1H 2027. | |
| Zhimeng Biopharma (private) | Enrolment complete in pivotal Phase 2/3 | CB03-154, potassium channel opener | ALS | ||
| Kidney | |||||
| Otsuka | Final Phase 3 VISIONARY results at ASN Kidney Week | Voyxact (sibeprenlimab), anti-APRIL | IgA nephropathy | Two-year eGFR stabilised; first reported 1 July. Moves that matter #2 | |
| Vera Therapeutics (VERA) | ORIGIN 3 final efficacy analysis at ASN; published in NEJM | Trutakna (atacicept), TACI-Fc | IgA nephropathy | Moves that matter #2 | |
| RemeGen; Vor Bio (VOR) | TELIGAN Phase 3 two-year kidney function data | Telitacicept, TACI-Fc | IgA nephropathy | eGFR slope -0.6 against -5.7 mL/min/1.73 m2 a year on placebo, p<0.001. China trial run by RemeGen. Moves that matter #2 | |
| Climb Bio (CLYM) | Initial PrisMN Phase 2 data at ASN | Budoprutug, anti-CD19 | Primary membranous nephropathy | 200 mg gave B-cell depletion, PLA2R antibody reduction and immunologic remission at 12 weeks. More data in 2027. | |
| Bone, skeletal and endocrine | |||||
| BridgeBio Pharma (BBIO) | Phase 3 CALIBRATE data at ASBMR | Encaleret, oral CaSR antagonist | Autosomal dominant hypocalcaemia type 1 | Bone turnover and participant-reported symptoms improved. FDA action date 8 May 2027. | |
| AstraZeneca (AZN), Alexion | HICKORY Phase 3 detail: primary endpoint missed | Efzimfotase alfa | Hypophosphatasia, treatment-naive adolescents and adults | 6-minute walk up 17.05 m against placebo at week 25, not significant. BLA under priority review. | |
| Regeneron (REGN) | OPTIMA extension: no new heterotopic ossification lesions | Pasatru (garetosmab), anti-activin A | Fibrodysplasia ossificans progressiva | Zero new lesions from week 56 to 84 in adults. Moves that matter #4 | |
| āshibio (private) | Part 1 preliminary efficacy, Phase 2/3 | Andecaliximab | Fibrodysplasia ossificans progressiva | 86.2% reduction in new flare-up rate. Moves that matter #4 | |
| Mirum Pharmaceuticals (MIRM) | Pivotal PROGRESS cohort 2 positive, ages 6 to under 12 | Atebrioz (zilurgisertib) | Fibrodysplasia ossificans progressiva | 25 patients. Approved for ages 12 and over in September; sNDA for younger children planned 1H 2027. Moves that matter #4 | |
| Ascendis Pharma (ASND) | Up to five years of kidney function data | TransCon PTH (palopegteriparatide) | Hypoparathyroidism, adults | Long-term follow-up, not a new primary readout. | |
| Lung | |||||
| Mediar Therapeutics (private) | Enrolment complete, Phase 2 WISPer | MTX-463, anti-WISP-1 | Idiopathic pulmonary fibrosis | 168 patients at more than 70 sites. Moves that matter #3 | |
| Endeavor BioMedicines (private) | Phase 2b WHISTLE-PF met primary endpoint | Taladegib (ENV-101) | Idiopathic pulmonary fibrosis | 213 patients, 73% on background therapy; ppFVC improved over placebo at 24 weeks. Effect size not disclosed. Moves that matter #3 | |
| Bristol Myers Squibb (BMY) | ALOFT-IPF Phase 3: lower dose met primary endpoint | Admilparant, LPA1 antagonist | Idiopathic pulmonary fibrosis | 60 mg met the FVC primary endpoint at week 52; 120 mg did not reach significance. About 1,255 patients. Moves that matter #3 | |
| Syndax Pharmaceuticals (SNDX) | MAXPIRe Phase 2 missed on FVC | Axatilimab, anti-CSF-1R | Idiopathic pulmonary fibrosis | Moves that matter #3 | |
| Calluna Pharma (private) | Dosing complete, Phase 2 AURORA | CAL101 | Idiopathic pulmonary fibrosis | Final independent safety review complete. Topline Q1 2027. Moves that matter #3 | |
| Liver, blood and metabolic | |||||
| BioMarin (BMRN) | Five-year Phase 3 extension data | Cipaglucosidase alfa-atga plus miglustat | Late-onset Pompe disease | ERT-experienced (n=62): walk up 0.7 and FVC down 2.8 points, % predicted, at year 5. ERT-naive (n=20): walk up 10.5 at year 4.5. | |
| Mirum Pharmaceuticals (MIRM) | Phase 3 EXPAND met primary endpoint | Livmarli (maralixibat) | Ultra-rare cholestatic liver diseases | Itch fell 1.81 points against 0.83 on placebo, p=0.0004; 52 patients. sNDA 1H 2027. | |
| Zydus Lifesciences (NSE: ZYDUSLIFE) | EPICS-III post hoc analysis by region at ACG | Saroglitazar | Primary biliary cholangitis | Treatment difference in biochemical response 60.7% in the US against 41.6% outside it. FDA action date 27 November. | |
| Rezolute (RZLT) | Enrolment complete, Phase 3 upLIFT | Ersodetug, antibody | Tumour hyperinsulinism | Topline guided to mid-December. | |
| Beren Therapeutics (private) | More than 10 years of preserved neurological function | Adrabetadex, cyclodextrin | Infantile-onset Niemann-Pick type C | FDA action date 17 November. Moves that matter #1 | |
| Disc Medicine (IRON) | Phase 3 APOLLO met all primary and secondary endpoints | Bitopertin, oral GlyT1 inhibitor | Erythropoietic protoporphyria | 183 patients. NDA resubmission planned by year end. | |
| Immunology and angioedema | |||||
| Pharvaris (PHVS) | Phase 3 RAPIDe-3 published in The Lancet | Deucrictibant, oral | Hereditary angioedema | Primary and all 11 secondary endpoints met; relief onset 1.28 hours. FDA action date 23 April 2027; MAA under EMA review. | |
| Zenas BioPharma (ZBIO) | New obexelimab data in IgG4-related disease | Obexelimab, CD19 and FcγRIIb | IgG4-related disease | ||
| Takeda (TAK) | Phase 2/3 IgGNITE results at ESID | TAK-881, subcutaneous immunoglobulin 20% | Primary immunodeficiency | Comparable to HyQvia with 50% less immunoglobulin volume. | |
| Pharming Group (PHAR) | Positive Phase II topline at ESID | Leniolisib, PI3Kδ inhibitor | Genetic PIDs with immune dysregulation beyond APDS | CVID Phase II topline due in November. | |
| Eye | |||||
| Innovent Biologics (HKEX: 1801) | Phase 3 RESTORE-3 met primary endpoint | Teprotumumab N01 (IBI311) | Thyroid eye disease | Proptosis response 60.4% against 23.5% at week 24 (p=0.0003); 116 patients in China. Already approved in China for TED. | |
| Sling Therapeutics (private) | Phase 2b/3 LIDS durability, post hoc | Linsitinib, oral IGF-1R | Thyroid eye disease | Of 20 responders, 95% held response to week 60 and 82% to week 72, off drug. Phase 3 ORBIT enrolling. | |
| Heart | |||||
| Longeveron (LGVN) | Updated ELPIS II Phase 2b results | Laromestrocel, cell therapy | Hypoplastic left heart syndrome | Company to request an FDA meeting; AHA presentation 8 November. | |
| Post hoc and real-world evidence | |||||
| BridgeBio Pharma (BBIO) | Independent real-world analysis favours acoramidis over tafamidis | Acoramidis (Attruby) | ATTR cardiomyopathy | 2,684 matched patients in Epic Cosmos; 18% lower risk of a composite of death, hospitalisation, new atrial fibrillation and diuretic intensification. | |
| Prilenia Therapeutics; Ferrer | Neurology paper links antidopaminergic drugs to clinical measures | Pridopidine | Huntington's disease | Published online 22 October. | |
- Discovery
- Clinic entry
- Readouts
- Regulatory
- Designations
- Access
- Deals
- Funding
Six approvals included the first complement inhibitor in IgA nephropathy, and Niemann-Pick type C gained a second pending filing
| Date | Company or institution | Event | Asset | Indication | Notes |
|---|---|---|---|---|---|
| Approvals | |||||
| Roche (ROG); Genentech | FDA approves Enspryng in thyroid eye disease | Enspryng (satralizumab), IL-6R inhibitor | Thyroid eye disease | First at-home subcutaneous treatment for TED, per Roche. Second indication after NMOSD; MOGAD filing under priority review. | |
| UCB (EBR: UCB) | FDA approves self-administered autoinjector | Zilbrysq (zilucoplan), peptide C5 inhibitor | Generalised myasthenia gravis, adults | Once-daily subcutaneous; a second self-administration option. | |
| BioCryst Pharmaceuticals (BCRX) | FDA approves 150 mg oral pellets | Orladeyo (berotralstat) | Hereditary angioedema prophylaxis, adults and adolescents | ||
| AstraZeneca (AZN), Alexion | FDA accelerated approval in IgA nephropathy | Ultomiris (ravulizumab), C5 inhibitor | IgA nephropathy, adults | First C5 complement inhibitor approved in IgAN, per AstraZeneca. UPCR reduced 43.4% at week 34. Moves that matter #2 | |
| Harbour BioMed (HKEX: 2142) | NMPA approves subcutaneous batoclimab | Batoclimab, anti-FcRn | Generalised myasthenia gravis | China approval. | |
| INOVIO Pharmaceuticals (INO) | FDA accelerated approval of VOCERYQ | VOCERYQ (INO-3107), DNA immunotherapy | Recurrent respiratory papillomatosis, adults | Approved to reduce the frequency of surgery. | |
| Europe and the UK | |||||
| Ultragenyx (RARE) | EMA: MAA validated | Rebisufligene etisparvovec (UX111) | MPS IIIA (Sanfilippo type A) | FDA approved on 17 September 2026 as FAYUVI. No EMA timetable given; Joint Clinical Assessment applies. | |
| Scholar Rock (SRRK) | EMA: MAA resubmitted with new fill-finish site | Apitegromab, anti-myostatin | Spinal muscular atrophy | First MAA withdrawn in August over GMP at a Catalent fill-finish site. FDA approved ISEMBYLD in September. CHMP opinion 1H 2027. | |
| Belite Bio (BLTE) | MAAs submitted in several markets through the Access Consortium | Tinlarebant, oral | Stargardt disease type 1 | US action date 12 February 2027. | |
| Ascendis Pharma (ASND) | CHMP: positive opinion | TransCon CNP (navepegritide), once weekly | Achondroplasia, children | Confirmed in the CHMP highlights for 12 to 15 October. | |
| Omeros (OMER) | CHMP: negative opinion confirmed on re-examination | Narsoplimab (Yartemlea), anti-MASP-2 | Transplant-associated thrombotic microangiopathy | ||
| INmune Bio (INMB) | Pre-MAA package filed with the MHRA | Ebstrocel, cell therapy | Recessive dystrophic epidermolysis bullosa | Potential UK approval guided to mid-2027. | |
| Filings and review timelines | |||||
| Nanoscope Therapeutics (private) | PMDA: NDA accepted with priority review | Sonpiretigene isteparvovec (MCO-010) | Inherited retinal dystrophies | Priority review via Sakigake designation. US BLA in retinitis pigmentosa under FDA review, no action date disclosed. | |
| Novo Nordisk (NVO) | FDA: BLA review extended; no new date | Denecimig, bispecific | Haemophilia A | Site remediation after a pre-licence inspection; no clinical deficiency cited. US launch now targeted for 1H 2027. | |
| BridgeBio Pharma (BBIO) | FDA: NDA accepted, priority review; PDUFA 4 Feb 2027 | Infigratinib, oral | Achondroplasia | PDUFA 4 February 2027. PROPEL 3: height velocity up 2.10 cm a year over placebo. EMA filing in Q4. | |
| Clene (CLNN) | NDA submitted to the FDA | CNM-Au8, oral nanocatalyst | ALS | ||
| Biohaven (BHVN) | FDA accepts NDA resubmission; advisory committee planned | Troriluzole (Vyglxia) | Spinocerebellar ataxia | Decision expected Q1 2027. Reported in Biohaven's Q3 release, 29 October. | |
| argenx (ARGX) | FDA grants priority review to paediatric sBLA | Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase) | Generalised myasthenia gravis, children | Action date February 2027. Reported with Q3 results. | |
| Palvella Therapeutics (PVLA) | FDA accepts NDA with priority review | QTORIN rapamycin, topical | Microcystic lymphatic malformations | Action date 28 February 2027. | |
| Rafael Holdings (RFL); Cyclo Therapeutics | NDA submitted on 28 October with a voucher request | Remibetadex (Trappsol Cyclo) | Niemann-Pick type C | TransportNPC missed its primary endpoint in the overall population. Moves that matter #1 | |
| Agios Pharmaceuticals (AGIO) | FDA sets a new action date for the sNDA | Mitapivat | Sickle cell disease | New action date 1 February 2027. | |
| FDA pathway and policy | |||||
| Wave Life Sciences (WVE) | FDA: FDA backs single registrational trial | WVE-006, GalNAc RNA editing | Alpha-1 antitrypsin deficiency | One two-year trial; a one-year interim could support accelerated approval. 600 mg monthly cohort data in Q4. | |
| Rezolute (RZLT) | FDA: Pre-BLA meeting recommended after Phase 3 miss | Ersodetug, antibody | Congenital hyperinsulinism | sunRIZE missed its primary and key CGM endpoints; the FDA gave no view on approvability. upLIFT topline this quarter. | |
| Benitec Biopharma (BNTC) | FDA: Type C meeting; pivotal design not agreed | BB-301, gene therapy | Oculopharyngeal muscular dystrophy | FDA asked for more data, including natural history. Final design in 1Q 2027; enrolment from mid-2027. | |
| Neurizon Therapeutics (ASX: NUZ) | FDA: HEALEY potentially the only registrational trial | NUZ-001, oral | ALS | Conditional on substantial evidence of effectiveness; NfL needs a strong rationale for accelerated approval. Topline late Q2 2027. | |
| Rocket Pharmaceuticals (RCKT) | FDA: Pivotal design aligned on 12 patients | RP-A501, AAV gene therapy | Danon disease | Twelve males; at least seven must meet LAMP2 and LV mass co-primaries at 12 months. Topline mid-2028. | |
| Orvida Pharma (private) | Accepted into the FDA Rare Disease Evidence Principles process | Ontivatrep (KM-023) | Olmsted syndrome | ||
| FDA | Launches the Rare Disease Framework Initiative | – | – | Led by the Rare Disease Innovation Hub with CBER and CDER, with a white paper, A New Framework for Rare Disease Review. Public meeting held 28 October; new or revised guidance may follow. | |
| Aardvark Therapeutics (AARD) | FDA lifts clinical hold | ARD-101, oral | Hyperphagia in Prader-Willi syndrome | Hold placed in May after ECG changes in a volunteer study. Restart depends on agreeing a Phase 3 design with the FDA. | |
- Discovery
- Clinic entry
- Readouts
- Regulatory
- Designations
- Access
- Deals
- Funding
Eight designations, from orphan status for an in vivo CAR-T in autoimmune haemolytic anaemia to EMA PRIME for Calico's fosigotifator
FDA, EMA and European Commission designations announced by the sponsor in October.
| Date | Company or institution | Event | Asset | Indication | Notes |
|---|---|---|---|---|---|
| FDA | |||||
| MavriX Bio (private) | FDA rare pediatric disease designation | MVX-220, AAV gene therapy | Angelman syndrome | AAV UBE3A gene therapy in Phase 1/2 ASCEND-AS across genotypes; voucher-eligible if approved. | |
| vTv Therapeutics (VTVT) | FDA orphan drug designation | HPP8668 (HPPD), oral | Sickle cell disease | First-in-class Nrf2 and Bach1 modulator; designation rests on sickle cell mouse model data. | |
| Nuevocor (private) | FDA Fast Track designation | NVC-001, AAV9 gene therapy | LMNA dilated cardiomyopathy | Granted as the first patient was dosed in the Phase 1/2 SUNBEAM-LMNA trial. | |
| Vivacta Bio; Grit Bio | FDA orphan drug designation | GT801, anti-CD19 in vivo CAR-T (mRNA in T-cell-targeted LNP) | Autoimmune haemolytic anaemia | Vivacta was spun out of Grit Bio's in vivo CAR-T platform. | |
| Octant (private) | FDA orphan drug designation | OCT-980 | Retinitis pigmentosa | ||
| EMA and European Commission | |||||
| Quoin Pharmaceuticals (QNRX) | EU orphan designation (EMA) | QRX003, topical | Peeling skin syndrome | Separate from Quoin's Netherton syndrome designations. | |
| Calico Life Sciences (private) | EMA PRIME designation | Fosigotifator, eIF2B activator | Vanishing white matter disease | ||
| Solid Biosciences (SLDB) | European Commission orphan designation | SGT-212, gene therapy | Friedreich's ataxia | FALCON trial continuing. | |
- Discovery
- Clinic entry
- Readouts
- Regulatory
- Designations
- Access
- Deals
- Funding
NICE backed two familial chylomicronaemia drugs in one month, and Canada moved on Spinraza and Lyvdelzi
| Date | Company or institution | Event | Asset | Indication | Notes |
|---|---|---|---|---|---|
| HTA and reimbursement | |||||
| NICE, England | Final draft guidance recommends olezarsen | Tryngolza (olezarsen), Sobi | Genetically confirmed familial chylomicronaemia syndrome | Announced by Sobi. | |
| Danish Medicines Council, Denmark | Recommendation kept and widened after price cut | Burosumab (Crysvita) | X-linked hypophosphataemia, children | New price negotiation. Earlier start in severe disease; young adults needing surgery covered for a limited period. From 1 January 2027. | |
| CDA-AMC, Canada | Draft recommendation supports wider public reimbursement | Spinraza (nusinersen), Biogen | Spinal muscular atrophy | Announced by Biogen Canada. | |
| pCPA, Canada | National price agreement reached | Lyvdelzi (seladelpar), Gilead | Primary biliary cholangitis | Announced by Gilead Canada; provincial listing is the next step. | |
| NICE, England and Wales | Plozasiran becomes a treatment option | Redemplo (plozasiran), Arrowhead | Familial chylomicronaemia syndrome, adults | Second NICE-backed FCS drug in October after olezarsen. | |
| Launches | |||||
| Zydus Lifesciences; MSN Laboratories | First US generic of Adempas launched | Riociguat tablets | Pulmonary arterial hypertension; CTEPH | ||
| Rhythm Pharmaceuticals (RYTM) | Japan launch after MHLW approval and NHI price listing | Imcivree (setmelanotide) | Acquired hypothalamic obesity | First approved therapy for the condition in Japan, per Rhythm. | |
| Takeda (TAK) | Simultaneous launch in Japan and China | Orzeyful (oveporexton), OX2R agonist | Narcolepsy type 1 | First orexin agonist available to prescribers. | |
| Egetis Therapeutics (STO: EGTX) | US launch | Emcitate (tiratricol) | MCT8 deficiency | ||
- Discovery
- Clinic entry
- Readouts
- Regulatory
- Designations
- Access
- Deals
- Funding
Shionogi's $2,000m for IntraBio led the month, and Ultragenyx sold two priority review vouchers for a combined $413m
Thresholds: any acquisition; licences with $25m or more upfront or $250m or more in total. Idorsia's Nxera deal (CHF 7m upfront) sits under Into the clinic. Ocugen's OCU400 licence in the Middle East and North Africa, Hiteck's NGF eye drop licence to Visara and Rumi's option on RTX-7340 disclosed no qualifying terms.
| Date | Company or institution | Event | Asset | Indication | Notes |
|---|---|---|---|---|---|
| Acquisitions | |||||
| Shionogi (TSE: 4507); IntraBio | Shionogi agrees to acquire IntraBio | AQNEURSA (levacetylleucine) | Niemann-Pick type C; ataxia-telangiectasia | $2,000m; no contingent payments disclosed. Closing planned November to December. Moves that matter #1 | |
| Alaunos Therapeutics (TCRT); Velaris Therapeutics | Alaunos acquires Velaris, a Paragon spinout | Velaris cardiopulmonary programmes | Pulmonary arterial hypertension | Paired with a $350m private placement. Acquisition terms undisclosed. | |
| BioMarin (BMRN); Alesta Therapeutics | BioMarin closes the Alesta acquisition | ALE1 | Hypophosphatasia | Closing announced by Alesta shareholder 1cBio. Terms undisclosed in that release. | |
| Ambrose Healthcare; AM-Pharma | Ambrose acquires AM-Pharma | AM-Pharma rare disease pipeline | – | Up to €161m in total; upfront undisclosed. Gives Ambrose a European base. | |
| Alcon (ALC); Neurotech Pharmaceuticals | Alcon acquires Neurotech | Encelto (revakinagene taroretcel) | Macular telangiectasia type 2 | $550m upfront. Encelto is the only FDA-approved MacTel treatment. | |
| Pelthos Therapeutics (PTHS); Castle Creek Biosciences | Pelthos to combine with Castle Creek | D-Fi (dabocemagene autoficel) | Dystrophic epidermolysis bullosa | 5.2m Pelthos shares (32.5%), up to 5.8m more shares and up to $300m in milestones. Close by end Q1 2027. | |
| Licensing and partnerships | |||||
| JCR Pharmaceuticals (TSE: 4552); Italfarmaco | Italfarmaco licenses pabinafusp alfa outside Japan | Pabinafusp alfa (JR-141), sold in Japan as IZCARGO | MPS II (Hunter syndrome) | US, Europe and Latin America. Upfront and total undisclosed; JCR keeps manufacturing. | |
| CSL (ASX: CSL); Alentis Therapeutics | CSL partners on lixudebart | Lixudebart, anti-claudin-1 antibody | ANCA vasculitis with RPGN; FSGS; PSC | Upfront $355m; total up to $1,555m, including up to $1,200m in commercial milestones. | |
| BioMarin (BMRN); GC Biopharma; Hanmi Pharm | BioMarin licenses a next-generation Fabry enzyme worldwide | HM15421/GC1134A, enzyme replacement | Fabry disease | Upfront $70m; total undisclosed. | |
| Priority review vouchers | |||||
| Ultragenyx (RARE) | Sells a rare pediatric disease voucher | Voucher from the GENGLYCOS approval | – | $210m sale price; buyer undisclosed. Voucher earned in GSDIa. | |
| Ultragenyx (RARE) | Sells a second voucher in a week | Voucher from the FAYUVI approval | – | $203m sale price; buyer undisclosed. Voucher earned in MPS IIIA. | |
- Discovery
- Clinic entry
- Readouts
- Regulatory
- Designations
- Access
- Deals
- Funding
Disc raised $450m two days after its Phase 3 win, and OMass closed a $310m Series C for adrenal disease
Rounds of $30m or more with a disclosed amount.
| Date | Company or institution | Event | Asset | Indication | Notes |
|---|---|---|---|---|---|
| Venture rounds | |||||
| RougeTx (private) | $58m Series A | RTX-001, once-daily oral small molecule | Hereditary haemorrhagic telangiectasia | BioGeneration Ventures, Angelini Ventures and Kurma Partners. Funds RTX-001 toward first-in-human dosing. | |
| Altay Therapeutics (private) | $37m Series A | DX5057, oral DUX4 inhibitor | FSHD | Takes the first oral DUX4 inhibitor into the clinic, per Altay. SOLVE FSHD portfolio company. | |
| OMass Therapeutics (private) | $310m Series C | OMS1620, MC2R antagonist | Congenital adrenal hyperplasia; Cushing's | Co-led by Bain Capital Life Sciences, Forbion and Novo Holdings. | |
| Star Therapeutics (private) | $200m private financing | Undisclosed preclinical programmes | – | Co-led by OrbiMed and Janus Henderson Investors. Takes preclinical programmes into the clinic and adds new ones. Incyte bought Star's Vega Therapeutics, with latarcibart (VGA039) for von Willebrand disease, in July for up to $2bn. | |
| Setara Biosciences (private) | $90m Series A | STAR-0871 (formerly ABS-0871), oral TRPV4 inhibitor | Charcot-Marie-Tooth disease type 2C | Deerfield, Canaan, Droia, EcoR1, Euclidean Capital and Regeneron Ventures; Jazz joined as a shareholder after buying Actio. Phase 1b THERON planned for 2027; discovery work in genetic epilepsies. | |
| Public and private placements | |||||
| Alaunos Therapeutics (TCRT) | $350m private placement alongside the Velaris acquisition | Velaris programmes | Pulmonary arterial hypertension | ||
| Disc Medicine (IRON) | Prices an upsized $450m public offering | Bitopertin | Erythropoietic protoporphyria | Upsized from $350m, two days after APOLLO. | |
Catalyst calendar
Six FDA action dates fall in November, opening with Beren’s NPC decision on 17 November and closing with Vertex in IgA nephropathy on 30 November
Exact dates come from the company, the regulator or the meeting organiser; month, quarter and half-year entries are company guidance. FDA action dates carry a slate bar.
| Timing | Company | Catalyst | Drug | Indication | What to look for |
|---|---|---|---|---|---|
| November 2026 | |||||
| 8 Nov | Longeveron (LGVN) | ELPIS II presentation, American Heart Association | Laromestrocel | Hypoplastic left heart syndrome | Whether the updated data support the FDA meeting Longeveron plans to request. |
| 9 to 12 Nov | EMA | CHMP meeting | – | – | Look for any rare-disease opinion a sponsor names in advance. |
| 17 Nov | Beren Therapeutics (private) | FDA action date | Adrabetadex | Infantile-onset Niemann-Pick type C | Extended three months after a major amendment. Look for the age range on the label. |
| 22 Nov | Savara (SVRA) | FDA action date | Molgramostim | Autoimmune PAP | Extended three months; no safety, efficacy or manufacturing concern cited, per Savara. |
| 25 Nov | Sanofi (SNY) | FDA action date, priority review | Venglustat | Gaucher disease type 3 | Look for label language on neurological manifestations. |
| 27 Nov | BridgeBio Pharma (BBIO) | FDA action date, priority review | BBP-418 | LGMD2I/R9 | Whether approval is full or accelerated. |
| 27 Nov | Zydus Lifesciences (NSE: ZYDUSLIFE) | FDA action date, priority review | Saroglitazar | Primary biliary cholangitis | EPICS-III: 56.7% biochemical response against 9.8% on placebo at week 52. |
| 30 Nov | Vertex (VRTX) | FDA action date, accelerated approval | Povetacicept | IgA nephropathy | Reviewed under a priority review voucher. Lands 40 days after the Ultomiris approval. |
| November | Pharming Group (PHAR) | Phase II CVID topline | Leniolisib | Common variable immunodeficiency | Guided to November in the Q3 release. |
| Nov to Dec | Shionogi (TSE: 4507) | IntraBio acquisition closes | AQNEURSA | NPC; ataxia-telangiectasia | Planned window. |
| December 2026 and the rest of the quarter | |||||
| Mid-Dec | Rezolute (RZLT) | upLIFT Phase 3 topline | Ersodetug | Tumour hyperinsulinism | Decides the BLA strategy after sunRIZE missed. |
| Dec 2026 | argenx (ARGX) | Phase 3 topline | Empasiprubart | Multifocal motor neuropathy | |
| By end 2026 | Disc Medicine (IRON) | NDA resubmission | Bitopertin | Erythropoietic protoporphyria | Built on the APOLLO data. |
| Q4 2026 | Wave Life Sciences (WVE) | RestorAATion-2, 600 mg monthly cohort data | WVE-006 | AATD | |
| Q4 2026 | BridgeBio Pharma (BBIO) | MAA submission | Infigratinib | Achondroplasia | |
| Q4 2026 | Satellos Bioscience | Phase 2 proof-of-concept start | Forazapadin | FSHD | IND cleared 24 September. |
| 2027 | |||||
| 21 Jan | Dyne Therapeutics (DYN) | FDA action date, priority review | Z-rostudirsen (DYNE-251) | Duchenne, exon 51 | |
| 1 Feb | Agios Pharmaceuticals (AGIO) | FDA action date, sNDA | Mitapivat | Sickle cell disease | Moved from the original date. |
| 4 Feb | BridgeBio Pharma (BBIO) | FDA action date, priority review | Infigratinib | Achondroplasia | Oral rival to Voxzogo and TransCon CNP. |
| 12 Feb | Belite Bio (BLTE) | FDA action date, priority review | Tinlarebant | Stargardt disease type 1 | |
| Feb 2027 | argenx (ARGX) | FDA action date, paediatric sBLA | Vyvgart Hytrulo | Generalised myasthenia gravis, children | |
| 28 Feb | Palvella Therapeutics (PVLA) | FDA action date, priority review | QTORIN rapamycin | Microcystic lymphatic malformations | |
| Q1 2027 | Biohaven (BHVN) | FDA decision after an advisory committee | Troriluzole (Vyglxia) | Spinocerebellar ataxia | |
| Q1 2027 | Calluna Pharma (private) | AURORA Phase 2 topline | CAL101 | IPF | |
| Q1 2027 | Benitec Biopharma (BNTC) | Final pivotal design confirmed | BB-301 | OPMD dysphagia | Enrolment planned for mid-2027. |
| End Q1 2027 | Pelthos Therapeutics (PTHS) | Castle Creek combination closes | D-Fi | Dystrophic epidermolysis bullosa | |
| Early 2027 | Bristol Myers Squibb (BMY) | ALOFT-PPF Phase 3 topline | Admilparant | Progressive pulmonary fibrosis | Tests whether 60 mg holds up outside IPF. |
| 23 Apr | Pharvaris (PHVS) | FDA action date | Deucrictibant | HAE attacks | MAA also under EMA review. |
| Early Q2 2027 | Coya Therapeutics (COYA) | ALSTARS Phase 2/3 topline | COYA 302 | ALS | |
| 8 May | BridgeBio Pharma (BBIO) | FDA action date | Encaleret | ADH1 | |
| Late Q2 2027 | Neurizon Therapeutics (ASX: NUZ) | HEALEY Regimen I topline | NUZ-001 | ALS | Potentially the only registrational study needed. |
| 1H 2027 | AstraZeneca (AZN), Alexion | FDA decision, priority review | Efzimfotase alfa | Hypophosphatasia | Priority review granted in September. |
| 1H 2027 | Vor Bio (VOR) | UPSTREAM MG Phase 3 topline | Telitacicept | Generalised myasthenia gravis | |
| 1H 2027 | Mirum Pharmaceuticals (MIRM) | sNDA submissions | Livmarli; Atebrioz | Rare cholestatic liver diseases; FOP ages 6 to under 12 | |
| 1H 2027 | Scholar Rock (SRRK) | CHMP opinion | Apitegromab | SMA | After the 8 October resubmission. |
| 1H 2027 | Novo Nordisk (NVO) | US launch target, pending FDA decision | Denecimig | Haemophilia A | No new action date set. |
| Mid-2027 | INmune Bio (INMB) | Potential UK approval | Ebstrocel | RDEB | |
| Mid-2027 | Rocket Pharmaceuticals (RCKT) | Pivotal dosing complete | RP-A501 | Danon disease | Topline guided to mid-2028. |
| 2027 | Climb Bio (CLYM) | More PrisMN Phase 2 data | Budoprutug | Primary membranous nephropathy | |
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