FDA
After laying off staff in 2022 to extend its cash runway, X4 Pharmaceuticals got its drug for the ultra-rare immunodeficiency disease across the finish line on Monday.
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After a leading study caused the FDA to slap its most stringent warning on hormone replacement therapies for menopause more than two decades ago, the regulator is changing course in what FDA Commissioner Marty Makary called a “historic day for women in the United States.”
The upheaval of the Health and Human Services workforce and leadership leaves much to be desired in terms of delivery, recently retired FDA Chief Information Officer Vid Desai tells BioSpace, but the regulatory agency is evolving to be more open to much needed change.
While the FDA continues to put out guidance documents and approve drugs, some companies are already reporting delays in dealings with the agency, while insiders warn of falling morale and a negative perception from the rest of the biopharma world.
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Three years after the FDA rejected its initial biologic license application, Mesoblast announced its resubmission for Ryoncil. Now, the treatment is getting a second chance.
BioMarin announced Tuesday that the FDA has accepted its supplemental New Drug Application to expand Voxzogo for children younger than five years with achondroplasia.
Wednesday, the FDA’s VRBPAC agreed that GSK’s respiratory syncytial virus vaccine candidate is safe and effective in older adults.
The FDA greenlit Reata Pharmaceuticals’ omaveloxolone (Skyclarys) as the first and only treatment for Friedreich’s ataxia, a rare, life-limiting neuromuscular disorder.
The hits kept coming for the sickle cell disease space last week as the FDA placed a full clinical hold on Fulcrum Therapeutics’ Investigational New Drug application FTX-6058.
The FDA approved Sanofi and Sweden-based Sobi’s efanesoctocog alfa, now marketed as Altuviiio, to treat bleeding in adults and children with hemophilia A.
There are currently no disease-modifying treatments for Friedreich’s ataxia. That could change when the FDA makes a decision about Reata Pharmaceuticals’ omaveloxolone on Feb. 28.
After overcoming a clinical hold, Astellas presented preliminary safety and efficacy data from the Phase I/II FORTIS trial of AT845 in late-onset Pompe disease at the 19th Annual WORLDSymposium 2023.
On Friday, the FDA approved Apellis Pharmaceuticals’ Syfovre (pegcetacoplan) as the first treatment for geographic atrophy (GA), a leading cause of blindness.
Takeda’s vedolizumab met its primary endpoint in the Phase III GRAPHITE study, significantly improving event-free survival in patients with acute graft-versus-host disease.