The CHMP Refused Nothing in September and Five Applications Were Pulled Anyway

Twelve positive opinions, no negative ones, and the coverage will count the twelve. The number that carries information is five: two initial applications and three indication extensions withdrawn before the committee ruled, one of them for a drug the FDA had approved six days earlier.

Apitegromab was approved in Washington on 11 September and withdrawn in Amsterdam by the 17th

The FDA approved Scholar Rock’s apitegromab, branded Isembyld, on 11 September 2026 for spinal muscular atrophy in adults and children aged two and over who are already receiving an SMN2-targeted treatment. It is the company’s first commercial product and the first muscle-targeted therapy in the disease.

Six days later the European application for the same molecule appears in the CHMP round as a withdrawal. Scholar Rock had told investors in March that the EMA review was progressing well with a decision anticipated in mid-2026.

The question and answer document explaining the withdrawal has not yet published, so the reason is not public. What is public is the sequence, and for anyone modelling European entry it is the sequence that matters. A withdrawal is not a refusal, but it resets the clock to a resubmission of unknown length rather than a Commission decision inside two months.

The same asymmetry sits underneath the Vertex filings. Two extensions covering children aged one year and older, for Kaftrio and Kalydeco, were pulled together. Withdrawing one paediatric extension is a file-specific decision. Withdrawing the matched pair on the same day is a programme decision.

The first biosimilar through the new pathway is a pertuzumab, six months after the paper was adopted

The reflection paper on a tailored clinical approach in biosimilar development, EMA/CHMP/BMWP/60916/2025, was adopted and published on 27 March 2026 after a consultation that ran from April to September 2025. Its operative sentence says comparative efficacy studies may not be required for well characterised biological substances where the analytical comparability exercise provides a more sensitive determination of biosimilarity, in conjunction with pharmacokinetic and appropriate safety studies.

Pebrilzo is the first product the committee has recommended following that approach. The EMA says so in the meeting highlights, which is an unusually direct piece of signalling for a document that normally lists products without commentary.

The choice of molecule is the part worth pausing on. A second pertuzumab biosimilar, Poherdy from Organon, received a positive opinion in February 2026 through the conventional route. So within seven months the same reference product has produced one biosimilar that ran a comparative efficacy trial and one that did not.

The comparative efficacy study has historically been the longest and most expensive element of a biosimilar programme, and the EMA’s stated aim is to reduce the clinical data required. Any originator model that assumes biosimilar entry is gated by the time needed to run a phase 3 equivalence trial in a sensitive indication now needs revisiting, and the effect is not limited to pertuzumab. It reaches every well characterised monoclonal antibody whose analytical package is strong, which is most of them. The immediate case is pointed: pertuzumab is the combination partner in the first-line Enhertu regimen that Japan approved on 16 September and that the CHMP has been reviewing in parallel. A regimen built on a branded backbone behaves differently once the backbone has two biosimilars pending decisions.

Novo won a new medicine and lost a paediatric extension in the same meeting

Frehemgo (denecimig) received a positive opinion for prophylaxis of bleeding episodes in haemophilia A. In the same round Novo Nordisk withdrew the application to extend Alhemo (concizumab) to children under 12 with haemophilia A or B, with or without inhibitors.

One company, one disease area, one meeting, opposite outcomes. Synopulse has reported previously that the first regulatory approval for denecimig came from Riyadh rather than Washington, and the European opinion now puts the molecule on a conventional path while the older asset’s paediatric expansion goes back to the drawing board.

The pattern across all three withdrawn extensions is the same. Every one of them was an attempt to move an authorised product into children. That is where European applications died quietly this month, and it is not visible in a headline that counts twelve approvals.

One count in the agency’s own statistics does not reconcile

The meeting highlights say the committee recommended extensions of indication for 11 medicines and name 11. The statistics panel on the same page reports 12 positive opinions on extensions of therapeutic indication for the month. The breakdown of new medicines is also partial, listing 7 non-orphan, 1 orphan and 2 biosimilars against a total of 12, with the 2 generics unaccounted for.

Neither gap changes the substance, and both probably reflect procedures counted separately from products. They are worth naming because the extension count is the figure most commonly lifted into competitive trackers, and the number in the panel and the number in the prose are not the same number.

What to watch

Commission decisions, which normally follow the opinion by about two months and will land in November for this cohort. The pending decisions include the first-line Enhertu combination and the teclistamab move into earlier lines, both of which change treatment position rather than adding a market.

Whether the tailored clinical approach appears again in October. One product is a signal. Two is a pathway, and the second is the one that tells biosimilar developers the route is open rather than exceptional.

The three re-examinations now running, for KemSu, Meplyffa and Qezzaqar. The committee confirmed its refusal of Xervyteg after re-examination this month, which is the base rate those three are working against.

Twelve approvals and no refusals reads as a generous month for Europe. Read the withdrawals instead and the month says something narrower: the committee did not have to refuse anything, because the applications that were going to fail left the room first.