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In an interview on Friday, FDA Commissioner Marty Makary threw his weight behind psychedelic therapies, noting that patients taking these substances experience significant benefits for various neuropsychiatric conditions.
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While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner Marty Makary’s proposal is light on details and raises potential concerns about safety, access and liability.
The FDA and NIH recently announced plans to phase out animal testing requirements for some therapies. While organoid and AI providers celebrate, scientists warn that questions over safety, applicability and implementation remain.
While the Trump administration has painted the jettisoning of staff and regulations as good for business, there are multiple reasons it’s unlikely to work out that way.
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With President Donald Trump expected to deliver a drug pricing order on Monday that Big Pharma and patient groups alike have railed against, the industry’s tumultuous ride is far from over.
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The restrictions on Novavax’s vaccine could portend changes at the FDA. Commissioner Marty Makary suggested last week that the agency could update its vaccine approval guidelines “in the coming days.”
Analysts at BMO Capital Markets said in a weekend note that a non-invasive blood test could help boost uptake of Alzheimer’s disease therapies.
The star of the acquisition is the enzyme replacement therapy INZ-701, being developed for the rare disease ENPP1 deficiency.
The FDA also approved the use of Zynyz as a monotherapy for patients with squamous cell carcinoma of the anal canal who are intolerant to platinum chemotherapy or whose disease has progressed.
Since Elevidys’ accelerated approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up.
Lilly will use Rznomics’ proprietary ribozyme technology to develop RNA editing therapies for congenital hearing loss.
Over Lars Fruergaard Jørgensen’s eight years as CEO, Novo’s sales, profits and share price have almost tripled, the company said. However, the shares have taken a turn since mid-2024, falling by half in one year.
As the FDA prepares for a busy Oncologic Drugs Advisory Committee meeting next week, an agency insider told BioSpace that volunteers with little training are scrambling to secure the required expertise after workforce cuts decimated the adcomm planning office.
After warnings that the dragged-out process was putting the cell therapy company at risk of bankruptcy, bluebird bio now has a new deal to offer shareholders.
The FDA also changed its tune and is now planning to convene an advisory committee to discuss Biohaven’s application.