Regulatory
In the five weeks since Donald Trump returned as U.S. president, the FDA, NIH and CDC have been thrown into disarray, with meetings regarding vaccines and rare diseases canceled or indefinitely postponed—all without a clear reason why.
The company will push through with an accelerated approval application for odronextamab in follicular lymphoma, leaving diffuse large B cell lymphoma behind.
The back-to-back high-level disruptions in vaccine policy under Robert F. Kennedy Jr. comes as the U.S. records its first death from measles since 2015.
Emalex is gearing up for a New Drug Application for ecopipam in Tourette syndrome later this year.
Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and upcoming drug volixibat.
Around 300 FDA staffers laid off last week are being asked to return. So far, the Trump administration has terminated some 1,000 employees from the agency.
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.
The postponed ACIP meeting comes barely a week after Robert F. Kennedy, Jr. was confirmed as Secretary of Health and Human Services, despite controversy regarding his anti-vaccine history.
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.
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