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The resubmission for RGX-121, expected in the third quarter, comes as the FDA has deemed REGENXBIO’s existing data “sufficient” to support an accelerated filing. It immediately follows a similar reversal of position regarding uniQure’s embattled Huntington’s disease gene therapy.
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If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
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?
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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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Gilead’s investigational drug combo bic/len could help lower the pill burden in patients with virologically suppressed HIV who are on complex treatment regimens, according to BMO Capital Markets.
A BioNTech spokesperson downplayed the news, insisting that the two companies remain “close” and have a “strong collaboration.”
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
Speaking at a conference this morning, Pfizer CEO Albert Bourla suggested that Metsera’s therapies could begin hitting the market in 2028.
Unpredictable communication and a lack of transparency are eroding the industry’s and the public’s trust. The FDA, experts agree, needs to take control of the narrative.
Korro Bio is moving back to square one as a preclinical biotech after the failure of KRRO-110 in alpha-1 antitrypsin deficiency. The company’s stock is down 80% on all the news.
The decision to pause dapiglutide will help Zealand focus investment into assets that have “the greatest potential for clinical differentiation” in obesity.
In January, AbbVie and Calico’s fosigotifator failed to show significant signs of efficacy in the HEALEY ALS platform trial for amyotrophic lateral sclerosis.
Alixorexton’s Phase II performance sets it up for late-stage success, according to analysts at Truist Securities. Alkermes expects to launch a global late-stage program early next year for narcolepsy type 2.
The plausible mechanism pathway “could accelerate gene therapy/editing development,” analysts at William Blair said Thursday, while adding that additional clarity is needed.