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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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Following promising Phase IIa data, Vertex Pharmaceuticals will now evaluate its oral drug candidate inaxaplin in a late-stage study of APOL-1 mediated kidney disease.
Following a series of rejections and clinical failures, Eiger BioPharmaceuticals has declared bankruptcy and will sell all its assets as the company winds down operations.
A federal appeals court Monday backed Teva and Viatris’ challenge to a lower court ruling, finding that the companies can again make their case against Johnson & Johnson’s patent covering its schizophrenia drug Invega Sustenna.
Alongside the abrupt departure of Chinese biotech WuXi from BIO, Congress is rattling sabers over perceived national security implications of the U.S.’s R&D relationship with the country.
While some companies look to AI models to stop revenue leakage in business operations, experts say more fundamental issues must first be addressed.
Fresh from its IPO, Fractyl Health got an Investigational Device Exemption from the FDA, clearing a pivotal study of its Revita system to help maintain weight loss following the discontinuation of GLP-1 drugs.
AstraZeneca has secured the FDA’s green light for Voydeya, a first-in-class, oral, Factor D inhibitor developed as an add-on to its standard-of-care Ultomiris or Soliris for certain patients with paroxysmal nocturnal haemoglobinuria.
The filing of a Biologics License Application for a subcutaneous version of Biogen and Eisai’s Leqembi (lecanemab) has been delayed due to procedural reasons, the companies announced Monday.
Despite strong demand for weight-loss drugs, the lack of Medicare coverage is potentially interfering with prescription and dispensing rates of anti-obesity medications for elderly adults, according to a new study.
Bristol Myers Squibb’s $4.8 billion acquisition of Mirati pays off with strong data from the KRYSTAL-12 study of Krazati, showing that the KRAS inhibitor significantly improves progression-free survival.