The FDA’s busy March continues with six target action dates remaining on the calendar. Over the next two weeks, the regulator will decide on investigational therapies for Duchenne muscular dystrophy, chronic kidney disease anemia, a rare metabolic disorder and more.
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Yesterday’s approval comes months after Novartis turned its back on tislelizumab, returning global rights to BeiGene. The companies signed a collaboration contract for tislelizumab in January 2021, but ran into several regulatory roadblocks.
The drug was approved under the accelerated approval pathway, which allows for earlier approval of drugs that treat serious conditions and address an unmet medical need, based on a surrogate or intermediate clinical endpoint that is reasonably likely to predict clinical benefit.
The FDA approved Bristol Myers Squibb’s Breyanzi for chronic lymphocytic leukemia and small lymphocytic leukemia prior to Friday’s adcomm for the company’s other CAR-T therapy, Abecma.
The California-based biotech also announced that it has filed a supplemental New Drug Application (sNDA) seeking approval for a higher concentration formulation of Livmarli for younger patients with progressive familial intrahepatic cholestasis (PFIC), a rare genetic disorder that causes progressive liver disease and can lead to significant morbidity and mortality. Mirum hopes to introduce this higher-concentration regimen later this year, according to its announcement.
In a briefing document for Thursday’s advisory committee meeting, the FDA pointed to efficacy and safety issues with Geron’s New Drug Application for imetelstat in myelodysplastic syndromes.
Staff reviewers said that the pattern of early deaths observed in patients treated with both the therapies in their respective trials raised uncertainty about the treatments’ overall survival benefit in patients.
The injectable drug imetelstat was being studied in patients with lower risk myelodysplastic syndromes (MDS), a type of blood cancer, which requires frequent blood transfusions for managing anemia.
Yesterday the agency published a revised draft guidance document, aimed at helping sponsors and pharmaceutical companies to develop therapies for Alzheimer’s disease, particularly in its early stages before the onset of overt dementia.
The U.S. FDA has declined to approve Viatris and Mapi Pharma’s once-a-month injection for treating relapsing forms of debilitating neurological condition multiple sclerosis (MS), the companies said on Monday.