EMA’s CHMP recommends three new orphan drugs, rebuffs two FDA-approved programs

At its February meeting, CHMP recommended six new medicines, including three orphan drugs already approved by the FDA. But CHMP also departed from the U.S. regulator, recommending one therapy the FDA had issued a complete response letter for and two the agency previously declined to approve. The recommendations include an mRNA vaccine from Moderna, a contrast with the FDA’s recent refuse-to-file U-turn on the company’s standalone mRNA flu application.

The three newly recommended orphan drugs that already have U.S. approvals are Ojemda tovorafenib for pediatric low-grade glioma (FDA 2024), Palsonify paltusotine for acromegaly (FDA 2025), and Xolremdi mavorixafor for warts, hypogammaglobulinemia, immunodeficiency, myelokathexis (WHIM) syndrome (FDA 2024). ...