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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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A suit against Novartis and Vitaris by Henrietta Lacks’ estate hinges on questions about the morality and legality of using the line for biopharmaceutical research.
While expected and seen as largely incremental, Jefferies analyst Peter Welford in a Tuesday note to investors said the detailed data for three early-stage assets support moving them into Phase IIb studies and creates a “foothold” for AstraZeneca in the weight loss space.
Coming off of a strong third quarter, Vertex Pharmaceuticals is nearing several important milestones, including the potential approvals of vanzacaftor triple in cystic fibrosis and the non-opioid therapy suzetrigine in pain—both slated for January 2025.
In a deal worth up to $285 million initially for the lead program, Novo Nordisk will gain access to Ascendis’ TransCon technology platform in an effort to find novel GLP-1 candidates with reduced dosing frequency.
Driven by the early approval of its updated COVID-19 vaccine, BioNTech far exceeded analysts’ expectations in the third quarter and reported its first quarterly profit in 2024. However, the German biotech also cut its outlook for the year.
Multiple players are exploring whether modalities designed to combat B cell malignancies can be repurposed against lupus, myasthenia gravis and other conditions traced to misdirected immune response.
The Big Pharma companies made a last-ditch effort asking a U.S. appeals court to reconsider their lawsuits against the Inflation Reduction Act’s Medicare drug price negotiations, which they contend infringe on their constitutional rights.
Jefferies analyst Roger Song in an investor note said that Viking Therapeutics’ readout for its investigational therapy VK2735 exceeded expectations, with “class-leading” weight loss. Patients on 100-miligram doses of the pill lost 8.2% of their body weight after 28 days.
The FDA is set to decide on four promising therapies in the next two weeks, including a CAR T for acute lymphoblastic leukemia.
This year has seen several biopharma companies drop Alzheimer’s and Parkinson’s disease programs, but experts say plenty are still chasing these multi-billion-dollar markets.