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After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
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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.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
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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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Eli Lilly on Monday announced a $3.2 billion all-cash deal to purchase Morphic and its pipeline of oral integrin therapies in a move to expand the pharma’s presence in the autoimmune diseases space.
Roche’s tiragolumab, when combined with its PD-L1 blocker Tecentriq, did not significantly improve progression-free and overall survival versus Keytruda and chemotherapy in patients with non-small cell lung cancer.
Eli Lilly, Rivus Pharmaceuticals and more target different biological processes in hopes of generating higher-quality weight loss and avoiding metabolic issues.
Two CRLs from the FDA last week cited concerns with third-party manufacturers, while Indian CDMOs may make a bid for U.S. business if there is a decoupling from Chinese companies under the BIOSECURE Act.
President Joe Biden and Sen. Bernie Sanders in a Tuesday op-ed in USA Today called on Novo Nordisk and Eli Lilly to “stop ripping off Americans” with “unconscionably high prices” for their GLP-1 medicines.
Johnson & Johnson and Legend Biotech’s Carvykti cell therapy significantly improved survival in patients with multiple myeloma when used in the second-line setting, the companies announced on Tuesday.
GSK on Wednesday restructured its contract with CureVac to gain access to the biotech’s influenza and COVID-19 programs for $430 million upfront and up to $1.13 billion in future payments.
Despite an uncertain legislative path to becoming law, the BIOSECURE Act has already impacted the biopharma industry’s confidence in Chinese service providers and prompted efforts to diversify manufacturing partners.
Taysha Gene Therapies looks to move past its pipeline culls and staff reductions by moving its lead asset into a possible billion-dollar market.
As congressional pressure increases on WuXi AppTec and other China-based companies over alleged ties to the Chinese government, India’s contract development and manufacturing organization sector could benefit.