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As uniQure prepares its closely watched Huntington’s disease gene therapy for FDA review, the biotech is also making waves in chronic epilepsy with another gene therapy that Stifel says is off to a “promising start.”
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While drugmakers and other stakeholders want to see faster approvals, experts say the FDA’s Commissioner’s National Priority Voucher program is still bereft of important details, with candidate selection and interference from the agency’s senior leaders topping the list.
UniQure’s planned third-quarter submission for its Huntington’s disease gene therapy may be a harbinger of a more flexible FDA under acting commissioner Kyle Diamantas—but how long will it last? And how can companies be sure these positive decisions won’t just be reversed?
While agents like AbbVie’s Humira have transformed the treatment landscape, not all patients benefit equally from the drug. Better biomarker analysis and more investment in mechanistic trials can inform the development of more effective therapies with broader clinical value.
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FDA veteran Peter Marks will now shape the future of Eli Lilly’s vaccines work after the buys of Curevo, LimmaTech Biologics and Vaccine Company for up to $3.8 billion total.
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Going private could give Recordati strategic flexibility and a stable source of capital, according to CVC Capital Partners and Groupe Bruxelles Lambert, which are offering to take the Italian pharma private for a 13% premium.
The agreement is the largest in a series of deals that Gilead Sciences has penned with Korea’s Yuhan.
Biogen and Denali’s Parkinson’s disease drug failed to significantly slow disease progression in a Phase 2b study, missing both primary and secondary endpoints.
After getting slapped with a surprise refuse-to-file letter signed by former CBER Director Vinay Prasad, Moderna’s flu vaccine application will now go before the FDA’s Vaccine advisory committee.
Update: Reversal after 20 deaths allows new Japan patients to take Amgen’s rare disease drug Tavneos
Kissei Pharmaceutical is reversing a recommendation related to Amgen-shared Tavneos that it made just a few days ago, now saying the rare disease drug can be given to new patients.
By partnering with a UN-backed body, Roche has enabled companies to make the medicine for supply in 129 countries.
Lilly met analysts’ sky-high expectations with 28.3% weight loss over 80 weeks for the triple hormone receptor agonist retatrutide in a highly anticipated readout on Thursday.
In September last year, a group of concerned stockholders raised alarm about Vaxart’s proposed reverse stock split, which the biotech was pushing for despite strong opposition from shareholders.
A trifecta of newly inked tech partnerships—from Eli Lilly, Bristol Myers Squibb and Incyte—exemplify the increasingly central role that AI is playing in drug development.
After being hit by safety issues and subpar results in another trial, BioMarin’s Phase 3 test of Voxzogo for a rare skeletal disorder called hypochondroplasia showed efficacy “solidly above” what the drug has shown for achondroplasia, which causes dwarfism.