JCR Gets Its Brain-Penetrating Hunter Therapy Out of Japan for the First Time, Through the UAE
IZCARGO, a therapy engineered to cross the blood-brain barrier, won its first marketing authorization outside Japan five years after launching there.
Rare disease
Crysvita Reaches One-Month-Old Infants in the EU, and Buys Two More Years of Orphan Exclusivity
The European Commission approved Crysvita for infants as young as one month with XLH, a decision that also buys Kyowa Kirin two more years of orphan exclusivity.
A Father’s Liver, One Incision: the First Single-Port Robotic Liver Transplant Lands in a Six-Year-Old
A six-year-old with a rare genetic liver disorder became the first patient to receive a liver transplant through a single robotic incision, donated by his father.
OrphanPulse Wk 29: Ionis Takes SMA-Proven Antisense Into Dravet Syndrome
A week defined by first-in-human starts across rare genetic disease, led by antisense entering Dravet syndrome from the field that already rewrote spinal muscular atrophy. Around it, first patients dosed in rare vascular and cardiomyopathy programs with near-empty competitive fields, a first-in-class readout in heparin-induced thrombocytopenia, and orphan designations that quietly reshape the economics behind them.
Reimbursement Series: CMS and Europe Reimbursement Pathway Accelerates Innovation
In a transformative week for market access, the CMS and Europe reimbursement pathway expansion delivered several pivotal coverage and pricing decisions reshaping patient access across cardiovascular, hepatic, and oncology therapies. From CMS’s national determinations on breakthrough devices to European HTA approvals in rare disease and cancer, these developments underscore a growing transatlantic alignment between regulators … Read more
Alexion Strikes Bold AI Partnership for Rare Disease Detection
AI partnership targets rare bone disease affecting 1 in 6,000 patients.
Bain Capital Acquires Mitsubishi Tanabe Pharma
Bain Capital, a leading global private investment firm with approximately $160 billion in assets under management, has announced its agreement to acquire Mitsubishi Tanabe Pharma Corporation (MTPC), a renowned Japanese pharmaceutical company with over 300 years of heritage. This transaction represents one of Bain Capital’s largest investments in the pharmaceutical sector and demonstrates its continued … Read more
NanoVation Therapeutics Partners with Novo Nordisk to Develop Genetic Medicines for Cardiometabolic and Rare Diseases
Overview of the Deal:NanoVation Therapeutics has entered into a multi-year partnership with Novo Nordisk to develop cutting-edge genetic medicines that target cardiometabolic and rare diseases. Announced on September 18, 2024, this collaboration will leverage NanoVation’s long-circulating lipid nanoparticle (lcLNP™) technology, which enables RNA delivery to cells outside the liver, combined with Novo Nordisk’s expertise in … Read more