CHMP backs Joenja for APDS, moving Pharming closer to EU approval

Pharming said the European Medicines Agency’s Committee for Medicinal Products for Human Use, or CHMP, has issued a positive opinion recommending authorization of Joenja (leniolisib) for activated phosphoinositide 3-kinase delta syndrome, or APDS, in adults and children ages 12 and older. If the European Commission follows that recommendation, Joenja would become the first approved treatment for APDS in the EU. The opinion follows a long review that was delayed in 2024 over manufacturing and chemistry, manufacturing, and controls questions, even as CHMP had already indicated the drug’s clinical benefit was positive. The application is backed by a randomized Phase II/III trial in 31 patients and long-term extension data in 37 patients with a median of three years of exposure. (stocktitan.net)

Why it matters: For veterinary professionals, this is another marker of how precision medicine is moving deeper into rare immunology, with regulators increasingly willing to back targeted therapies for genetically defined syndromes. While APDS is a human indication, the bigger signal is translational: pathway-specific immunology drugs are gaining traction, and that can shape expectations around diagnostics, specialty referrals, and future comparative research across species. The commercial picture also matters. Joenja is already approved in the U.S., has NICE-backed access in England and Wales, and Pharming has been expanding internationally, suggesting the company is building a broader footprint around rare immune dysregulation. (globenewswire.com)

What to watch: The next step is a European Commission decision, which Pharming said is expected in Q2 2026. (stocktitan.net)