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The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
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With the biopharma industry performing better of late, analysts, executives and other industry watchers are “cautiously optimistic”—a term heard all over the streets of San Francisco at the J.P. Morgan Healthcare Conference earlier this month.
Bristol Myers Squibb, GSK and Merck are contributing drug ingredients as part of their deals with the White House but are keeping many of the terms of their agreements private.
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.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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The approvals come as Robert F. Kennedy Jr.—who has been critical of vaccines—takes leadership of the Department of Health and Human Services.
Ono picked up Romvimza—previously known by its active ingredient vimseltinib—from its $2.4 billion acquisition of Deciphera Pharmaceuticals in April 2024.
Merilog’s approval comes as the insulin space has over the past year suffered several setbacks, including strong calls for price caps and, potentially, the rise of the mammoth GLP-1 market.
Continuing our SCOPE 2025 coverage, Rohit Nambisan, CEO at Lokavant addresses not only current challenges, but the life sciences industry’s responsibility to maintain scientific integrity.
After the rejection of Lykos Therapeutics’ MDMA-based PTSD treatment tempered excitement for psychedelic therapeutics, a recent approval and positive data are generating new momentum, which experts expect to continue throughout 2025 and 2026.
Biopharma doubles down on immunology and inflammation as companies target new pathways and seek to improve on current options in inflammatory bowel disease, atopic dermatitis, myasthenia gravis and more.
With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this disease space alongside Avidity Biosciences, Lupin and AMO Pharma.
The company, helmed by BioNTech alums, is developing therapies aimed at dermatological, respiratory and gut-related indications.
In its fourth-quarter earnings report, Moderna’s revenue was down substantially from 2023. Separately, media reports reveal anticipated cuts to the company’s digital team.
The failure in adjuvant melanoma could cause BMS and Opdualag to miss out on a market opportunity that is nearly twice as large as its current approved indication, according to analysts.