In an article that reads like informal guidance, U.S. FDA Commissioner Marty Makary and CBER Director Vinay Prasad discussed the criteria for using the agency’s novel plausible mechanism pathway for personalized treatments when a randomized trial isn’t feasible, as well as future uses of the approval path that could expand beyond gene and cell therapies to other biologics and even small molecules.
In reaching within to name 26-year FDA veteran Richard Pazdur as the next CDER director, FDA Commissioner Marty Makary likely chose the best possible person to helm the drug evaluation center at a time marked by uncertainty, morale issues and upheaval, according to several agency watchers.
The U.S. House is expected to vote late Nov. 12 on an amended continuing resolution (CR) to end the historic 43-day partial government shutdown. Already passed by the Senate, the CR would fully reopen the government and fund it through Jan. 30. President Donald Trump has said he will sign the CR, which ensures federal employees furloughed during the shutdown will receive back pay and will not be terminated.
The U.S. FDA is turning the clock back more than 20 years to advance women’s health by narrowing the boxed warning on hormone replacement therapies (HRTs) for menopause. The agency announced at a Nov. 10 news conference that it’s working with companies to update their HRT labeling to remove references to risks of cardiovascular disease, breast cancer and probable dementia.
Industry watchers were surprised when Eli Lilly and Co.’s oral GLP-1 candidate, orforglipron, did not appear among the first nine recipients of the U.S. FDA’s commissioner’s national priority voucher (CNPV) program aimed at shortening regulatory review times, boosting domestic manufacturing and improving affordability. But orforglipron, which recently nailed endpoints in a second phase III trial and has been hailed a potentially best-in-class compound, was among the second batch of six drugs added to the CNPV list.
The U.S. FDA’s complete response letter (CRL) for Biohaven Ltd.’s lead asset, troriluzole, to treat spinocerebellar ataxia has prompted a wave of downstream changes at the company. There will be a roughly 60% cutback in annual R&D spending, not including personnel, as Biohaven focuses on three other late-stage clinical programs.
The U.S. FDA has approved UCB SA’s Kygevvi (doxecitine and doxribtimine), the first treatment for the ultra-rare, genetic and life-threatening mitochondrial disease thymidine kinase 2 deficiency. The approval comes as the company reaps a reward of rebuilding and reorganizing that it began little more than a year ago.
Uniqure NV is regrouping after a surprise switcheroo by the havoc-beset U.S. FDA regarding phase I/II studies with AMT-130 vs. external control in Huntington’s disease (HD) – news of which pushed down the Lexington, Mass.-based firm’s shares (NASDAQ:QURE) Nov. 3 by $33.40, a loss of 49%, at the closing price of $34.29.
The U.S. FDA once again has a leadership gap at the top of its drug center, which already has been ravaged this year by massive terminations, resignations and retirements of senior leaders. George Tidmarsh, a biopharma industry veteran who’s helmed CDER for a little more than three months, resigned effective immediately Nov. 2 after being placed on administrative leave two days earlier amid a Department of Health and Human Services probe.
Celltrion Inc. announced Oct. 29 the signing of an $87.75 million joint drug R&D agreement with AI and spatial transcriptome-based biotech Portrai Inc.
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