FDA
Neither Chongqing Qiancan Technology nor Tianjin Healthcare Medical Technology provided the records requested by the FDA, leading the agency to issue import alerts and warning letters.
FEATURED STORIES
Inclusion of the patient perspective is currently an “optional” part of the rare disease drug development process, CBER Director Karim Mikhail told BioSpace. Mikhail, along with his CDER counterpart Michael Davis and other agency rare disease leaders, is trying to change that.
Also lined up are target action dates for Sanofi’s Gaucher disease therapy, GSK’s lung cancer medicine and BridgeBio’s muscular dystrophy drug.
By allowing compounding pharmacies to produce certain unapproved peptides, the Department of Health and Human Services could, in the short term, put patients in harm’s way. Longer term, the policy shift threatens the industry that brings new medicines to market.
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Twelve marketed drugs, including Eli Lilly’s and Novo’s weight loss mega-blockbusters, will require a label change under the new FDA update.
If approved, the oral GLP-1 would be Eli Lilly’s second marketed drug for obstructive sleep apnea after the GIP/GLP-1 injectable Zepbound secured the first approval in the space in December 2024.
Vir Biotechnology is targeting a mid-2027 regulatory submission for its monthly chronic hepatitis delta regimen, with head-to-head data against Gilead’s Hepcludex expected next quarter.
Compass Pathways has completed an FDA filing for COMP360, with an expected launch in the first half of next year. The update follows a reported regulatory submission for Usona Institute’s own psilocybin candidate for depression last Monday.
After Novartis confirmed the reports of tumors in patients who have been treated with the spinal muscular atrophy gene therapy, the FDA will determine if any regulatory action is needed for Zolgensma or for the pharma’s similar product Itvisma.
After the FDA delayed a decision for Agios Pharmaceuticals’ sickle cell disease candidate, the biopharma is letting go of more than 35% of its R&D employees.
The majority of the large pharmas have now reported third quarter earnings—spoiler alert: Eli Lilly made more money than anyone expected. Plus, the FDA dropped another initiative aimed at accelerating rare disease treatments; HHS greenlights the compounding of six unapproved peptides; and uniQure’s embattled gene therapy for Huntington’s disease has finally been accepted for review.
After a year of controversial regulatory reversals, the FDA has accepted uniQure’s application for ifezuntirgene inilparvovec for priority review, with a May 1 action date. If approved, the therapy—also called AMT-130—would be the first disease-modifying treatment for Huntington’s disease.
Former biologics boss Peter Marks had a vision. With the FDA’s latest draft guidance, it’s coming to fruition. Speaking with BioSpace, current CBER leader Karim Mikhail said the “adapted pathway” is designed to develop personalized gene therapies more quickly.
If approved, BridgeBio Pharma’s BBP-418—which, according to one Jefferies analyst, boasts “pretty darn compelling” data—would be the first therapy for limb-girdle muscular dystrophy. The FDA has an action date of Nov. 27.