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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The approval marks the first time a Korean company independently brought a drug from discovery to FDA approval.
The U.S. Food and Drug Administration approved Alnylam Pharmaceuticals’ Givlaari (givosiran) for acute hepatic porphyria.
AbbVie’s blockbuster drug Humira just got another challenger to its share of the market in the United States when its patent expires in 2023.
“The approval of Adakveo marks a new era in the treatment of sickle cell disease, a genetic condition that places an extraordinary burden of unpredictable pain crises on patients and their families,” said Susanne Schaffert, president of Novartis Oncology.
It is the first BeiGene-discovered drug to be approved.
The U.S. Food and Drug Administration approved Shionogi’s Fetroja as a treatment for adult patients with complicated urinary tract infections caused by a number of Gram-negative microorganisms.
The approval marks the first approved treatment in the United States for this condition.
Ziextenzo (pegfilgrastim) is a long-acting version of filgrastim and is indicated to decrease the incidence of infection in cancer patients.
In clinical trials, Talicia demonstrated 90% efficacy in the eradication of H. pylori in studies and is the only rifabutin-based therapy on the market.
The approval was based on a New Drug Application (NDA) submitted under the 505(b)(2) filing pathway.