Amsterdam: The Committee for Medicinal Products for Human Use met from 14 to 17 September and came out with twelve positive opinions on new medicines and eleven extensions of indication for products already on the market. None of those decisions authorises anything yet. They are recommendations, and they now travel to the European Commission, which normally takes two to three months to convert an opinion into a marketing authorisation valid across all member states.
The interval between opinion and authorisation is one of the least understood features of European medicines regulation. To a patient reading a headline, a drug has been approved. To a hospital pharmacist, nothing has changed and will not change until the Commission decision issues, national pricing negotiations conclude, and a reimbursement body decides whether the product represents value at the price asked. That last stage is entirely outside the agency’s remit and varies enormously between member states. A medicine recommended in September may reach patients in one country within months and in another only after years, or not at all.
Among the September opinions, several address conditions where the existing options are narrow. Frehemgo, with the active substance denecimig, is intended for preventing bleeding episodes in haemophilia A, the inherited disorder caused by a deficiency of factor VIII. It works by mimicking the activity of factor VIIIa rather than replacing the missing protein, and is delivered in a pre-filled pen with dosing intervals that can be weekly, fortnightly or monthly. For a condition historically managed by frequent intravenous infusion, the shift in administration burden is the clinically interesting part.
Gevalka, containing ensartinib, received a positive opinion for adults with anaplastic lymphoma kinase positive advanced non-small cell lung cancer, a molecularly defined subgroup where targeted agents have progressively displaced conventional chemotherapy. Klygefa, with gefurulimab, is directed at generalised myasthenia gravis, an autoimmune condition producing muscle weakness and fatigue. Lifyorli, containing relacorilant, is intended for platinum-resistant epithelial ovarian, fallopian tube or primary peritoneal cancer, an indication where response rates to existing treatment have remained disappointing.
The eleven indication extensions attract less attention but often matter more in aggregate. Extending an authorised medicine to a new patient population, a younger age group or an earlier line of therapy can enlarge the treated population far more than a novel product with a narrow indication. It is also the mechanism through which accumulated post-authorisation evidence feeds back into the label.
The committee’s output this year sits against a legislative backdrop that is still unsettled. The revision of the general pharmaceutical legislation, proposed in 2023, remains in negotiation between the Parliament and the Council, with the duration of regulatory data protection and the conditions attached to it the most contested element. Separately, the Critical Medicines Act reached a provisional political agreement on 12 May 2026 and awaits formal ratification by both institutions, with publication in the Official Journal expected towards the end of the year. That instrument approaches supply security from the procurement side, requiring resilience criteria such as stockholding obligations and diversification of supply sources to be weighted in public tenders rather than leaving price to dominate.
Whether procurement criteria can do what industrial policy has not is an open question. The manufacturing base for older off-patent medicines, including several antibiotics and intravenous generics, has consolidated into a small number of sites concentrated outside Europe, and it consolidated because the margins made anything else uneconomic. Writing resilience into tender documents raises the price the public purchaser pays. That is the intended effect, and it is also the reason the instrument will be tested hardest by the member states with the tightest health budgets.
The twelve opinions from this month will work through the system on their own timetable, largely unaffected by any of it.
