CHMP backs Joenja for APDS, setting up EU decision

Pharming said the European Medicines Agency’s Committee for Medicinal Products for Human Use, or CHMP, has issued a positive opinion recommending EU approval of Joenja (leniolisib) for activated phosphoinositide 3-kinase delta syndrome, or APDS, in adults and children ages 12 and older. The recommendation follows a long review that had been delayed by a remaining chemistry, manufacturing, and controls question, even as CHMP had already affirmed the drug’s clinical benefit and safety. Joenja is already approved in the U.S., and has also secured reimbursement support in England and Wales through NICE; Pharming now says a final European Commission decision is expected in the second quarter of 2026. (stocktitan.net)

Why it matters: APDS is an ultra-rare primary immunodeficiency, estimated to affect roughly 1 to 2 people per million, and it can present with recurrent infections, lymphoproliferation, autoimmunity, and increased lymphoma risk. For clinicians, an EU authorization would expand access to the first disease-specific therapy for a condition that often requires complex long-term management, including immunoglobulin replacement, anti-infective support, immunosuppression, and sometimes transplant consideration. The decision also signals that Pharming has cleared the regulatory hurdle that previously centered on manufacturing, not the underlying efficacy or safety package. (ncbi.nlm.nih.gov)

What to watch: The next milestone is the European Commission’s final decision, which typically follows a positive CHMP opinion within about two months and, if granted, would cover all EU member states. (stocktitan.net)