FDA
Congressional letters sent to the CEOs of Eli Lilly, Pfizer, Merck, BMS and AbbVie this week voicing concerns about the pharmas’ clinical trials in China highlight an ongoing discrepancy in how government and industry think about the rise of the Asian country’s biotech industry.
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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.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
When the variance can’t be modeled, even disciplined biotech investors stop deploying. Here’s the cheapest fix for biotech’s investability problem.
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Pemgarda has a standing emergency use authorization as a prophylaxis for immunocompromised patients, but FDA’s stringent requirements for antibody activity boxed out its potential use as a post-exposure treatment.
Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and upcoming drug volixibat.
Many of these unlawful and unauthorized shipments were explicitly tagged for compounding, according to a new analysis. Separately, a group of state attorneys general has raised concerns about the unsafe GLP-1 drugs finding their way to American consumers.
Morale is low at the FDA, which was hit with layoffs this week following RFK Jr.’s confirmation. Biopharma leaders and agency insiders fear further workforce cuts could delay new medicines.
Despite the regulatory setback, analysts appear optimistic, noting that Harmony’s long-term prospects remain bright given the development of its high-dose formulation of its sleep-regulating drug Wakix.
The FDA is mired in uncertainty with some staffers losing their jobs over the weekend and more potentially to come, vaccines and psychedelic therapies could be facing very different futures under newly confirmed HHS Secretary Robert F. Kennedy, Jr., Moderna continues its downward revenue slide and Merck, Regeneron, BMS and more face strong patent headwinds.
The hammer came down on an unspecified number of FDA employees this weekend, days after Robert F. Kennedy Jr. was confirmed as HHS Secretary.
The approvals come as Robert F. Kennedy Jr.—who has been critical of vaccines—takes leadership of the Department of Health and Human Services.
Ono picked up Romvimza—previously known by its active ingredient vimseltinib—from its $2.4 billion acquisition of Deciphera Pharmaceuticals in April 2024.
Merilog’s approval comes as the insulin space has over the past year suffered several setbacks, including strong calls for price caps and, potentially, the rise of the mammoth GLP-1 market.