FDA
While a new facility setup program aimed at encouraging onshoring received a positive reception at a recent meeting, industry representatives said the current rules on existing production plants are the main regulatory issues facing manufacturing teams.
FEATURED STORIES
On the FDA’s docket for the back half of September is Merck’s proposed subcutaneous formulation of its blockbuster cancer drug Keytruda.
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.
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From more than 30 target action dates in the last three months of the year, BioSpace has narrowed the list to six regulatory decisions that could have far-reaching implications for biopharma and patients.
Phase III data showed that Inluriyo improves progression-free survival versus standard endocrine therapy.
The regulatory greenlight was backed by two Phase III trials that showed normalized growth hormone levels in patients with the rare pituitary condition. It’s the first approval for Crinetics Pharmaceuticals and something CEO Scott Struthers predicted “will transform people’s lives.”
Three draft recommendation documents published on Wednesday are intended to guide drug sponsors and accelerate the development of cell and gene therapies.
The regulatory action marks the second rejection for a spinal muscular atrophy therapy this week after Scholar Rock’s apitegromab was issued a complete response letter on Tuesday, similarly on manufacturing grounds.
In its rejection letter, the FDA flagged problems at a third-party fill-finish site owned by Novo Nordisk. Issues at this site have previously been investigated by the regulator.
The agency also pointed to the use of Tylenol and other acetaminophen products during pregnancy as being potentially linked to neurological and developmental defects in children, following a press conference Monday in which President Donald Trump did the same.
An advisory committee last year found that Zynquista’s benefit-risk profile in type 1 diabetes was unfavorable due to cases of diabetic ketoacidosis.
The company was expecting a decision from the FDA by Sept. 28 for its oral drug tolebrutinib, but an update to the drug’s application package convinced the agency to take more time to review.
For the last two years, Keytruda has reigned as the world’s top-selling drug—a distinction under threat with key patent protections expiring in 2028.