FDA
If approved, ensifentrine would be the first non-steroidal, anti-inflammatory drug for patients with chronic obstructive pulmonary disease, offering an option with potentially fewer side effects.
FEATURED STORIES
New FDA expert panels, such as recent meetings on SSRI use during pregnancy and on hormone replacement therapy during menopause, are drawing criticism for being one-sided. One leader says such panels are designed to reach a specific conclusion.
Amid an unprecedented turnover in leadership at the FDA and mass layoffs of staff, communication has crumbled and uncertainty runs rampant, leaving small and medium biopharma companies without a clear path forward for their therapies.
Aside from the rare disease market, Novo Nordisk also scored a key regulatory win last month for its blockbuster GLP-1 drug Wegovy, which can now be used to treat patients with metabolic dysfunction-associated steatohepatitis.
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This week: Cancer license deals from J&J and BeiGene, a potential $7B acquisition by Roche and confirmed $1.9B Lilly buy, EU fine for Illumina, and more legal challenges to the Inflation Reduction Act
The regulator on Thursday approved Opill, the first oral contraceptive available in the U.S. without a prescription. Perrigo shares rose 6% in response to the news.
The FDA recently approved the first cellular therapy for Type 1 diabetes and others may not be far behind. But experts say challenges still exist to the widespread application of these treatments.
Thursday, Eisai and Biogen’s Leqembi also became the first disease-modifying treatment for Alzheimer’s to win traditional approval. CMS coverage is expected to begin immediately.
The Inflation Reduction Act could put an end to blockbuster runs like that of Merck’s Keytruda, experts told BioSpace. In the meantime, the drug keeps picking up more indications and positive clinical results.
After an initial rejection, BioMarin has finally secured the FDA’s approval for Roctavian, the first gene therapy in the U.S. for the most common form of the bleeding disorder.
Of the 30 patients given CellTrans’ Lantidra in two studies, 21 were insulin-free for at least a year and 10 were insulin-free for more than five years.
Overcoming an FDA rejection in January 2022, Pfizer and OPKO’s Ngenla will provide a long-acting, reduced-frequency treatment option for children with growth hormone deficiency.
The biopharma’s monoclonal antibody is the first to win approval for the two most common forms of generalized myasthenia gravis, a rare autoimmune and neuromuscular disorder.
Shares of Sarepta dropped 11% a day after securing accelerated approval for the first gene therapy to treat Duchenne muscular dystrophy over concerns about the potential for label expansion.