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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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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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The drugs’ active ingredient, glatiramer acetate, has been linked to more than 80 cases of anaphylaxis worldwide since December 1996 and six patient deaths.
The deal follows a $1.06 billion U.S. contract in July 2024 and a $1.24 billion agreement with an Asia-based pharma a few months later.
The U.S. Court of Federal Claims ruled that Vanda cannot claim a breach of confidentiality for specifications that it did not develop itself.
Cebranopadol, a dual-NMR agonist, reached the primary endpoint in a Phase III trial and matched placebo for safety, a significant concern in the analgesic field.
On the company’s Q4 earnings call where an eyepopping $88.8 billion in full-year sales were revealed, leaders shifted focus away from enormous takeovers to single-digit billion buy outs.
At J.P. Morgan, most biopharma executives expressed a neutral stance on the incoming administration, but just days later, President Trump issued multiple executive orders that concern the industry.
With an eye toward advancing a novel antibody-drug conjugate for gastrointestinal cancers, ArriVent is the latest biopharma player to ink a deal with a Chinese biotech.