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Roche said the Phase II results help build the case for advancing CT-388 into late-stage testing, which is set to get underway this quarter.
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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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After winning expanded approval for its gene therapy for Duchenne muscular dystrophy, Sarepta’s leadership and analysts see a sizeable commercial opportunity on the horizon.
It’s important to maintain anonymity when seeking your next executive role, as your departure could impact other employees and the organization.
Following success in several indications including ulcerative colitis, J&J’s blockbuster drug Tremfya failed to meet a mid-stage study’s primary endpoint in treating giant cell arteritis.
Citing issues with a third-party manufacturer, the FDA has issued another Complete Response Letter to AbbVie rejecting its New Drug Application for ABBV-951, a proposed treatment for motor fluctuations in adults with advanced Parkinson’s disease.
AstraZeneca’s PD-L1 inhibitor failed to significantly improve disease-free survival in patients with non-small cell lung cancer, but hit its primary endpoint in a late-stage trial in muscle-invasive bladder cancer.
If approved, ensifentrine would be the first non-steroidal, anti-inflammatory drug for patients with chronic obstructive pulmonary disease, offering an option with potentially fewer side effects.
After being spun off of sequencing giant Illumina, Grail on Tuesday is set to start trading on the Nasdaq Global Select Market following a years-long antitrust battle with regulators.
Merck KGaA’s drug candidate xevinapant in a late-stage trial was unable to significantly improve event-free survival in patients with locally advanced head and neck cancer.
Novo Nordisk on Monday announced it is boosting its manufacturing capabilities with a $4.1 billion commitment to construct a second fill and finishing facility in Clayton, North Carolina.
Exsilio Therapeutics emerged from stealth on Tuesday with a platform that leverages mRNA technology to develop redosable genomic medicines for a range of complex diseases.