FDA
Last month, the FDA launched TrialBlazer, intended to streamline the IND path and bring early clinical trials and medical innovation home to the U.S. It’s a start, but new agency leadership must see it through.
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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.
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The U.S. FDA had a very packed calendar for this week, but almost everything on it was under a Priority Review pathway.
Palovarotene is being developed for the prevention of heterotropic ossification, which is new bone formation in people with fibrodysplasia ossificans progressive.
The amended EUA is aimed at patients who have undergone solid organ transplants or those diagnosed with conditions that have an “equivalent level of immunocompromised.”
This approval was based on the Phase III withdrawal study that showed clinically meaningful differences vs. placebo in change to the primary endpoint, Epworth Sleepiness Scale score.
The U.S. FDA greenlit the combination of Keytruda and Eisai’s Lemvima as a first-line treatment for adult patients with advanced renal cell carcinoma.
Sanofi has announced that the U.S. FDA approved its new treatment for children one year of age or older diagnosed with late-onset Pompe disease.
Although August is a fairly busy month for PDUFA dates, there were only two on the U.S. FDA’s calendar for this week, and one of those has already been reported. Here’s a look.
Sierra will have access to AZD5153, a potent and selective BRD4 BET inhibitor, to initiate a study that combines the said component with momelotinib for treating patients with myelofibrosis.
Aprea faces another disappointment this week in the clinic. The FDA held up a yellow card, and the Boston-based biotech has placed a partial clinical hold on its myeloid malignancy program.
Eisai said that they have initiated communication with the FDA to achieve “the most optimal regulatory pathway” for lecanemab.