Nexletol and Nexlizet for LDL Lowering and CV Risk, Iclusig for ALL, Elahere for Ovarian Cancer, Duvyzat for DMD, Spevigo for Psoriasis, Tryvio for Hypertension, Opsynvi for PAH, Lenmeldy for Juvenile Metachromatic Leukodystrophy

New FDA Approvals

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• Nexletol and Nexlizet for LDL Lowering and CV Risk: The FDA approved bempedoic acid (Nexletol) and its combination with ezetimibe (Nexlizet) for reducing cardiovascular risk and treating primary hyperlipidemia. Bempedoic acid inhibits a cholesterol synthesis enzyme, while ezetimibe blocks cholesterol absorption, both lowering LDL-C levels. The approval, for high-risk patients not yet having cardiovascular events, was based on the CLEAR Outcomes trial.
• Iclusig for ALL: The FDA granted accelerated approval to ponatinib (Iclusig) with chemotherapy for treating previously untreated Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Ponatinib, a tyrosine kinase inhibitor, is the first targeted agent for this leukemia type in combination with chemotherapy, based on the PhALLCON trial results. Further confirmatory data may be required to verify its clinical benefit.
• Elahere for Ovarian Cancer: Mirvetuximab soravtansine-gynx (Elahere) received FDA approval for treating folate receptor alpha-positive, platinum-resistant ovarian cancer, marking it as the first antibody-drug conjugate in the U.S. for this condition. The treatment targets cancer cells by binding to the folate receptor alpha, delivering a cytotoxic agent. Approval was based on the Phase 3 MIRASOL trial.
• Duvyzat for DMD: The FDA approved givinostat (Duvyzat) for patients 6 years or older with Duchenne muscular dystrophy (DMD), a rare neuromuscular condition. Givinostat is an HDAC inhibitor that mitigates muscle damage and slows disease progression. Approval was based on the EPIDYS trial results, granted to Italfarmaco S.p.A.
• Spevigo for Psoriasis: Spesolimab-sbzo (Spevigo) received FDA approval for treating generalized pustular psoriasis (GPP) in adults and pediatric patients, expanding its indication from initial approval for GPP flares. As the first targeted therapy for GPP, it acts as an IL-36 receptor antagonist. The expanded approval was based on the Effisayil 2 trial, showing significant reduction in GPP flares.
• Tryvio for Hypertension: The FDA approved aprocitentan (Tryvio) in combination with other antihypertensive drugs for adults not adequately controlled on other medications, marking it as the first new oral antihypertensive therapy pathway in nearly 40 years. Based on the PRECISION trial, aprocitentan was shown to be effective in patients with resistant hypertension.
• Opsynvi for PAH: Macitentan and tadalafil (Opsynvi) was approved by the FDA for adults with pulmonary arterial hypertension (PAH) and WHO functional class II-III, as the first once-daily single-tablet combination therapy for PAH. The approval was based on the A DUE study, demonstrating greater reduction in pulmonary vascular resistance compared to monotherapies.
• Lenmeldy for Juvenile Metachromatic Leukodystrophy: The FDA approved Lenmeldy (atidarsagene autotemcel) gene therapy for children with metachromatic leukodystrophy (MLD), a rare genetic disease affecting the brain and nervous system. The therapy uses the patient's own genetically modified stem cells to produce the missing enzyme. Approval was based on significant improvements in survival, mobility, and cognitive functions observed in clinical trials and an expanded access program.