Norgine Secures CHMP Backing for First EU Therapy for WHIM Syndrome Under X4 Deal

New updates have been reported about Norgine.

Norgine has gained a key regulatory boost with the European Medicines Agency’s CHMP issuing a positive opinion recommending marketing authorisation under exceptional circumstances for XOLREMDI (mavorixafor) to treat WHIM syndrome in patients aged 12 and older, positioning the company to launch the first authorised WHIM therapy in Europe. The European Commission is expected to issue its final decision in the second quarter of 2026, a timing that will shape Norgine’s launch and market access planning across its European footprint.

Under a January 2025 licensing and supply agreement, Norgine will assume full responsibility for commercialising mavorixafor in Europe, Australia and New Zealand once approvals are in place, with all marketing authorisations in those territories to be transferred to Norgine and X4 Pharmaceuticals supplying the product. Backed by phase 3 4WHIM trial data in 31 patients demonstrating efficacy and safety, the product strengthens Norgine’s rare and specialty medicines portfolio and could open a new revenue stream in an ultra‑rare immunodeficiency where no other licensed treatment exists, while leveraging the company’s regional infrastructure and market access capabilities to maximise uptake.