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Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a confirmatory trial.
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At the BIO International Convention in San Diego, attendees marked the 50th anniversary of original biotech Genentech, reflecting on the immense challenges facing companies as China becomes a powerhouse innovator.
A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.
Recent approvals for Corcept Therapeutics and Merck have injected momentum into the space, where GSK, Allarity Therapeutics, OSE Immunotherapies and others are advancing their own candidates.
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If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
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Analysts at BMO Capital Markets expect the lack of other exon-44-skiping therapies to facilitate a “smooth” approval process for Avidity’s del-zota.
Claiming that the domestic market undervalues pharma innovation, Merck has decided to pull the plug on all of its R&D efforts in the U.K.
The autoimmune and inflammatory disease–focused company canceled plans to go public earlier this year as the IPO window slammed shut.
As Novo Nordisk cuts 9,000 people from its organization in a restructuring effort, BioSpace looks back on the Danish pharma company’s rise.
These 27 markets, comprising countries across Asia, Europe and South America, together contributed some 12% of Lundbeck’s earnings in 2024.
Jefferies analysts expect a regulatory filing for rocatinlimab later this year, with a product launch in 2026.
Cullinan Therapeutics and Taiho Oncology’s zipalertinib elicited promising response rates in two mid-stage studies of non-small cell lung cancer patients with typical and uncommon EGFR mutations.
Former CDC director Susan Monarez and former chief medical officer Debra Houry will appear in front of the Senate HELP Committee on Sept. 17.
Novo Nordisk also lowered its full-year profit growth guidance in connection with the restructuring effort. The pharma now anticipates operating profit to grow from 4% to 10%, down from its prior projection of 10% to 16%.
This week’s release of the Make America Health Again report revealed continued emphasis on vaccine safety; Health Secretary Robert F. Kennedy Jr.’s faceoff with senators last week amounted to political theater; the FDA promises complete response letters in real time and shares details on a new rare disease framework; and Summit disappoints at the World Conference on Lung Cancer in Barcelona.