Myeloma Bispecific, Uveal Melanoma, HD Eylea, Huntington's, Fibrodysplasia Ossificans Progressive, CHAPLE Disease

New FDA Approvals

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· Elrexfio (elranatamab-bcmm) has received accelerated approval for the treatment of adult patients with relapsed or refractory multiple myeloma who have undergone multiple prior therapies. Elrexfio is a subcutaneously delivered bispecific antibody (BsAb) immunotherapy activating T-cells to kill myeloma cells. Approval is contingent upon verification of clinical benefit in further trials. The approval was granted to Pfizer. Elrexfio joins other bispecific antibody therapies from Janssen for the same condition.

· The FDA has approved the Hepzato Kit for delivering melphalan to the liver as a treatment for metastatic uveal melanoma (mUM) with unresectable hepatic metastases. This is the only liver-directed therapy approved for this purpose. Approval was based on the FOCUS Study (NCT02678572). The manufacturer, Delcath Systems, intends to explore broader applications beyond uveal melanoma due to the therapy's potential for other tumor types.

· A higher-dose version of Eylea (aflibercept) injection has been FDA-approved for treating wet age-related macular degeneration (wAMD), diabetic macular edema (DME), and diabetic retinopathy (DR). This approval is based on PULSAR and PHOTON pivotal trials demonstrating non-inferior vision gains at longer dosing intervals compared to the standard dose. Eylea is a VEGF inhibitor, approved for wet AMD in 2011. The approval was granted to Regeneron Pharmaceuticals.

· Ingrezza (valbenazine) capsules have gained FDA approval for treating chorea associated with Huntington's Disease (HD). The approval is supported by clinical studies and was granted to Neurocrine Biosciences. Ingrezza was previously approved for tardive dyskinesia.

· The FDA has approved Sohonos (palovarotene) to treat fibrodysplasia ossificans progressive (FOP), a rare bone disease causing heterotopic ossification. The approval is based on Phase 3 MOVE trial results, showing effective reduction in bone growth. Sohonos is selective for a subtype of retinoic-acid receptors. It received Orphan Drug and Breakthrough Therapy Designations and is manufactured by Ipsen.

· Veopoz (pozelimab-bbfg) has gained FDA approval as the first treatment for CD55-deficient protein-losing enteropathy (CHAPLE disease). CHAPLE disease is a rare inherited immune disorder causing complement hyperactivation. Veopoz is a complement inhibitor indicated for patients aged 1 year and older. It received multiple designations and was granted approval to Regeneron Pharmaceuticals.

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