Chiesi Group’s idebenone faced a regulatory setback last month after the U.S. FDA issued a complete response letter (CRL) to the company’s NDA for Leber hereditary optic neuropathy (LHON), a rare inherited disorder that causes sudden vision loss.But a chance missed for Chiesi may be an opportunity for gene therapies, including Gensight Biologics SA’s lenadogene nolparvovec (Lumevoq; GS-010).
Wegovy (semaglutide) has passed the notoriously strict cost-effectiveness scrutiny of the U.K. health technology assessment body and will now be reimbursed for the prevention of further serious cardiovascular events in people who have had a heart attack or stroke, or are diagnosed with peripheral arterial disease.
Deep molecular advances are enabling precision medicine for the field of hematology, Wyndham Wilson said during a plenary session at the 2026 Korean Society of Hematology International Conference March 26.
Eli Lilly and Co. anticipates shipping newly approved Foundayo (orforglipron) within the next week, as the drug becomes the second oral weight-loss glucagon-like peptide-1 (GLP-1) receptor agonist to enter the U.S. market following December’s approval of Novo Nordisk A/S’ Wegovy (semaglutide) pill.
Braveheart Bio Inc. reported positive results from an open-label randomized phase II dose-ranging study of BHB-1893 (HRS-1893) in obstructive hypertrophic cardiomyopathy. Results presented at the American College of Cardiology’s Annual Scientific Session and Expo in New Orleans showed BHB-1893 treatment was associated with rapid and substantial reductions in left ventricular outflow tract gradient, the primary endpoint of the study.
The U.S. path forward is narrowing for Vanda Pharmaceuticals Inc.’s tradipitant as a treatment for gastroparesis, a serious disorder for which there’s been no new treatment in several decades.
Biogen Inc. hardly blinked at the competition faced by Apellis Pharmaceuticals Inc. as the two companies sealed a deal whereby the former has agreed to acquire all outstanding shares of the latter for $41 each, or about $5.6 billion.
Eli Lilly and Co. plans to buy Centessa Pharmaceuticals plc for $6.3 billion in up-front cash and another potential $1.5 billion through contingent value rights, gaining access to a pipeline of orexin receptor 2 agonists for sleep disorders. The Indianapolis-based pharma is re-entering a field that has multibillion-dollar potential and one that could emulate the success seen with its obesity program. Centessa, of Boston and London, has a lead candidate, cleminorexton (formerly ORX-750), with positive phase IIa data in narcolepsy types 1 and 2 and idiopathic hypersomnia.
Otsuka Pharmaceutical Co. Ltd. is taking a neuroplastogen approach to posttraumatic stress disorder (PTSD) through its planned $1.22 billion acquisition of Transcend Therapeutics Inc. The deal gives Tokyo-headquartered Otsuka access to Transcend’s lead asset, TSND-201, an oral neuroplastogen that has begun patient recruitment for a phase III study in the U.S.
Eli Lilly and Co. is deepening its investment in artificial intelligence-driven drug discovery through a multibillion-dollar expansion of its collaboration with Hong-Kong listed Insilico Medicine Inc. in a move that could broaden its reach into next-generation metabolic therapies.
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