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The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
After last year’s ‘stampede’ for FGF21 assets, the focus for the metabolic dysfunction-associated steatohepatitis space has shifted toward differentiated approaches, such as THR-β agonists and combination treatments, that seek to mirror the commercial success of Madrigal’s Rezdiffra.
Maintaining America’s momentum demands that policymakers resist policies that undermine research and development incentives.
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FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
FDA
Following the FDA’s refusal to review Moderna’s investigational mRNA flu vaccine last week, Commissioner Marty Makary faced questions from the U.S. president about the agency’s handling of vaccines. It’s a clear signal that the tension long brewing at the drug regulator has now gone all the way to the top.
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Judge Zahid Quraishi on Thursday appeared unconvinced by the pharmaceutical companies’ arguments that the Inflation Reduction Act’s Drug Price Negotiation Program violated the U.S. Constitution.
FDA
BeiGene’s Brukinsa becomes the first BTK inhibitor approved for follicular lymphoma, the most common type of low-grade non-Hodgkin lymphoma.
Gilead Sciences is partnering with Dutch biotech Merus to find novel dual tumor-associated antigens that target tri-specific antibodies.
The antibody-drug conjugate beat the standard of care in second-line multiple myeloma, the company reported on Thursday, as it seeks to get the cancer drug back on the U.S. market.
The FDA’s busy week ahead involves three decision dates for potential industry firsts and a highly anticipated advisory committee meeting for two CAR-T therapies.
Novo Nordisk’s early-stage amylin and GLP-1 co-agonist elicited a 13.1% reduction in body weight, with an overall favorable safety profile, the Danish drugmaker reported Thursday at an investor event.
The Anglo-Swedish drugmaker is making the investment to bolster its vaccine manufacturing and research and development capabilities in the U.K.
President Joe Biden has proposed expansions to the Inflation Reduction Act, including an increase in the annual number of prescription drugs that would be subject to price negotiations to 50, up from 20.
The early-stage oncology startup filed for an initial public offering on Wednesday, seeking funds to help develop its novel cancer therapies targeting extrachromosomal DNA.
The recently launched company shared two posters at the Muscular Dystrophy Association’s annual conference this week ahead of a March 21 PDUFA date.