FDA
The FDA issued a rare Refusal-to-File letter to Moderna over its mRNA-based influenza vaccine application, in an unusual move that sent the biotech’s shares tumbling.
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The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding for the Immigration and Customs Enforcement’s large-scale crackdown in Minnesota and other states.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
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AbbVie’s antibody-drug conjugate Elahere on Friday won the FDA’s full approval for the treatment of FRα-positive, platinum-resistant ovarian, fallopian tube and primary peritoneal cancers.
Duvyzat, which will be sold in the U.S. by ITF Therapeutics, joins a growing market that includes recently approved gene therapy Elevidys and corticosteroid Agamree.
Months after Johnson & Johnson turned its back on the hypertension treatment Tryvio, Idorsia has secured the FDA’s nod for the endothelin receptor blocker.
The recent FDA decision will likely mean more Medicare patients gain access to the blockbuster weight loss drug, experts say. Meanwhile, results continue to roll in for GLP-1 agonists for conditions beyond diabetes and obesity.
Takeda on Tuesday secured another label expansion for the kinase inhibitor, this time in the first-line setting for the treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia.
Orchard Therapeutics on Monday secured the FDA’s first approval for an autologous gene therapy to treat the rare metabolic disease metachromatic leukodystrophy in children.
With its FDA approval last week and first-to-market advantage, Madrigal Pharmaceuticals’ Rezdiffra will set the standard for other metabolic dysfunction-associated steatohepatitis candidates in development.
By votes of 11-0 and 8-3, respectively, an FDA advisory committee Friday deemed the risks of early death for both Johnson & Johnson’s Carvykti and Bristol Myers Squibb’s Abecma acceptable.
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.
Despite skepticism from FDA reviewers, the Oncologic Drugs Advisory Committee on Thursday strongly supported Geron’s imetelstat for the treatment of anemia in patients with lower-risk myelodysplastic syndromes.