CHMP backs Joenja for rare immune disorder APDS in Europe

Pharming said the European Medicines Agency’s Committee for Medicinal Products for Human Use, or CHMP, has issued a positive opinion recommending approval of Joenja (leniolisib) for activated phosphoinositide 3-kinase delta syndrome, or APDS, in adults and children ages 12 and older. The March 27 recommendation follows a prolonged EU 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 in 2024. If the European Commission follows the recommendation, as it often does, Joenja would become the first approved APDS treatment in the EU. Pharming said a final decision is expected in the second quarter of 2026. (stocktitan.net)

Why it matters: APDS is an ultra-rare primary immunodeficiency linked to recurrent infections, immune dysregulation, and long-term complications including bronchiectasis. EMA orphan-drug materials have estimated the EU patient population at roughly 500 people, and noted that no satisfactory EU-approved treatments were available at the time of designation, with care centered on supportive measures such as immunoglobulin replacement and infection control. For clinicians, an EU authorization would add a targeted oral PI3Kδ inhibitor to a space that has largely relied on symptom management rather than disease-specific therapy. (ema.europa.eu)

What to watch: The key next step is the European Commission decision, expected in Q2 2026, and whether broader access in Europe is followed by payer and national reimbursement moves. (stocktitan.net)