EMA Recommends Rare Blood Cancer Treatment and Seven Other Medicines

The European Medicines Agency (EMA) announced on Nov. 10, 2023 that its Committee for Medicinal Products for Human Use (CHMP) has recommended approval of eight new medicines at its latest meeting. The medicines, three of which were designated as orphan medicines, include treatments for generalized myasthenia gravis, glioma, neovascular age-related macular degeneration, and more.

Omjjara (momelotinib), an orphan medicine, was recommended for treating the rare blood cancer, myelofibrosis, which affects the bone marrow.Another orphan drug, Rystiggo (rozanolixizumab), was recommended for approval to treat generalized myasthenia gravis, which is a chronic autoimmune neuromuscular condition. The third orphan drug, Spexotras (trametinib), was given a positive opinion for treating glioma, a brain tumor that develops in glial cells, in pediatric patients aged one year and older.

Two biosimilars and two generic drugs were also recommended for approval. The biosimilar Rimmyrah (ranibizumab) was recommended for treating the retinal macular disease, neovascular age-related macular degeneration. The second biosimilar, Uzpruvo (ustekinumab), was recommended to treat plaque psoriasis, psoriatic arthritis, and Crohn’s disease. The generic drugs that received a positive CHMP opinion include Azacitidine Kabi (azacitidine), for the treatment of myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia; and Naveruclif (paclitaxel), which treats metastatic breast cancer, metastatic adenocarcinoma of the pancreas, and non-small cell lung cancer.

CHMP also granted conditional marketing authorization after a re-examination to Krazati (adagrasib) for the treatment of advanced non-small cell lung cancer with a G12C mutation in the KRAS gene in adults whose disease has worsened after at least one systemic treatment. The committee also extended recommendations for nine previously authorized medicines: Ayvakyt (avapritinib), Evkeeza (evinacumab-dgnb), Fluad Tetra (influenza vaccine [surface antigen, inactivated]), Jardiance (empagliflozin), Keytruda (pembrolizumab), Mounjaro (tirzepatide), NexoBrid (anacaulase-bcdb), Talzenna (talazoparib), and Veltassa (patiromer).

Two applications for recommendation were withdrawn. Vijoice (alpelisib), an orphan drug, which would have been for the treatment of PIK3CA-related overgrowth spectrum, was withdrawn. Bylvay (odevixibat), also an orphan drug, had an application for extension of use to include treatment of cholestatic pruritus in Alagille syndrome in patients aged six months or older withdrawn.

Source: EMA