FDA
The FDA previously rejected Zycubo for Menkes disease in October last year, citing issues with the drug’s manufacturing facility.
The FDA initially placed the Phase III IDeate-Lung02 study on hold due to a “higher than expected” number of deaths in patients treated with ifinatamab deruxtecan.
Drugmakers told the FDA that inflexible post-approval change requirements are among the top regulatory barriers to the reshoring of pharmaceutical manufacturing.
FDA Commissioner Marty Makary called these changes “common-sense reforms” that could expedite the development of cell and gene therapies.
In his annual letter, Flagship Pioneering’s Noubar Afeyan lays out a choice between near-term “human-made miracles” and a reversion to the pain and suffering of past diseases due to “growing contempt” in the U.S. for the scientific method.
The DC-based biopharma disputed the FDA’s conclusions regarding the data provided in its supplemental application for Hetlioz and promised to keep pushing for an approval.
Novo Nordisk follows Christmas oral Wegovy approval with quick launch; Eli Lilly is headed for $94.3 billion in annual revenue by 2027, analysts predict; nine more pharmas strike Most Favored Nation deals but half remain unsigned; experts call for stability and rare disease action at FDA, and all eyes are on M&A ahead of the J.P. Morgan Healthcare conference next week.
Last month, the FDA declined to approve Sanofi’s tolebrutinib for a specific form of multiple sclerosis. In a recently published complete response letter, the agency detailed its reasoning behind the rejection.
Only a handful of the top pharmas have signed Most Favored Nation drug pricing deals with the White House, while smaller biotechs continue to hang in limbo.
In a year that saw advisory committees placed under a particularly bright microscope at the FDA, the agency held fewer meetings than usual and agreed with its advisors only 57% of the time, Jefferies reported.
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