The RNA drug’s accelerated approval brings Sarepta’s exon-skipping market share to nearly 30%.
Sarepta Therapeutics, Inc. gained another inch with the US Food and Drug Administration approval of its third drug for Duchenne muscular dystrophy, but the mile ahead may come in the form of next-generation therapies for DMD. For some of them, data could emerge even before confirmatory data arrives for the company’s approved first-generation drugs.
The FDA granted accelerated approval to the Cambridge, MA-based biotech’s Amondys 45 (casimersen) on 25 February, for DMD patients with...
Highlights from Day Four of the BIO International Convention include Woodcock offering practical advice on rare disease trials, the sorry state of dealmaking mid-year, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.
While some are calling for Syncona to be wound up, the UK-based company believes many existing and new investors will back the creation of new private fund.
Antibody-drug conjugates are a major part of the oncology pipeline, and data presented at the American Society of Clinical Oncology meeting highlight the emergence of new technologies.
Lilly’s Mounjaro for diabetes rose from sixth to third position among the top-selling drugs in the first quarter of 2025, Rival Ozempic retained second position and Merck & Co’s immuno-oncology blockbuster Keytruda stayed far ahead.
Lilly’s Mounjaro for diabetes rose from sixth to third position among the top-selling drugs in the first quarter of 2025, Rival Ozempic retained second position and Merck & Co’s immuno-oncology blockbuster Keytruda stayed far ahead.
Oral RNA splicing modulator has gone into a Phase II/III trial
Highlights from Day 3 of the BIO International Convention include the realities of MFN pricing, AstraZeneca's R&D plans for obesity, the need for resources for FDA's rare disease hub and reactions to the Commissioner's National Priority Review Voucher program.
