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The resubmission for RGX-121, expected in the third quarter, comes as the FDA has deemed REGENXBIO’s existing data “sufficient” to support an accelerated filing. It immediately follows a similar reversal of position regarding uniQure’s embattled Huntington’s disease gene therapy.
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If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
While drugmakers and other stakeholders want to see faster approvals, experts say the FDA’s Commissioner’s National Priority Voucher program is still bereft of important details, with candidate selection and interference from the agency’s senior leaders topping the list.
UniQure’s planned third-quarter submission for its Huntington’s disease gene therapy may be a harbinger of a more flexible FDA under acting commissioner Kyle Diamantas—but how long will it last? And how can companies be sure these positive decisions won’t just be reversed?
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FDA veteran Peter Marks will now shape the future of Eli Lilly’s vaccines work after the buys of Curevo, LimmaTech Biologics and Vaccine Company for up to $3.8 billion total.
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With one disease-modifying therapy already reaching patients and another expected to soon, several biopharma companies anticipate key data for novel assets in the coming 12 months.
While AbbVie beat revenue expectations in the fourth quarter of 2023, the company reported Friday that its overall sales last year declined as it faced biosimilar competition for its blockbuster Humira.
The pharma Friday reported in-line and new product revenue growth of 9%, bringing in $9.8 billion for the fourth quarter of 2023. Eliquis and Opdivo remain top sellers but BMS is facing the loss of exclusivity.
The British pharma expects “meaningful” sales and earnings growth in 2024 and is upgrading its growth outlooks for 2026 and 2031, according to GSK CEO Emma Walmsley.
The U.S. Department of Health and Human Services on Thursday released new data showing that Americans pay three times more for prescription drugs than other developed countries and nearly 10 times more for insulin.
Alto Neuroscience and Fractyl Health provided further momentum to the recent spate of biotech initial public offerings, with both companies going public on Friday morning in respective $128 million and $110 million IPOs.
Kyverna Therapeutics, the fifth biotech with plans for an initial public offering this year, will use the proceeds to support the development of its anti-CD19 CAR-T therapies for autoimmune diseases.
The Centers for Medicare and Medicaid Services on Thursday sent its opening price proposals to drugmakers, which now have 30 days to either approve the offers or submit counteroffers.
Biotech companies are exploring multiple targets in the fight against neuroinflammation. Experts say these projects could yield the next breakthrough in treating Alzheimer’s disease.
The Swiss pharma on Thursday projected mid-single-digit sales growth for 2024, culling eight assets and looking to acquisitions and collaborations for de-risked assets with “significant potential.”