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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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The reinstatement of the generic drug policy office is the latest reversal of course for Robert F. Kennedy Jr.'s HHS, which also recently rehired FDA staff responsible for making travel arrangements and those involved in user fee program negotiations.
Two weeks of upheaval at the CDC culminated Monday in the complete reconstitution of the CDC’s Advisory Committee on Immunization Practices as HHS Secretary Robert F. Kennedy Jr. pens op-ed criticizing “conflicts of interest” he says exist on the current committee.
Avidity has been given the go-ahead by the FDA to request accelerated approval of delpacibart braxlosiran—potentially the first disease-modifying treatment for facioscapulohumeral muscular dystrophy—with an application in the second half of 2026.
The loss of special government employee status for four members of the CDC’s Advisory Committee on Immunization Practices follows the resignation of ACIP co-lead Lakshmi Panagiotakopoulos, who left in protest of a controversial change in COVID-19 recommendations for healthy children and pregnant women.
After a major shareholder pushed back, Keros is returning half of its capital to investors in a move that Guggenheim analysts called “a positive step forward.”
Merck’s enlicitide decanoate significantly lowered low-density lipoprotein cholesterol in two Phase III trials, unlocking what BMO Capital Markets called a “multi-billion dollar opportunity” for the pharma.
In what Guggenheim Partners called one of Metsera’s “critical program milestones” this year, its ultra-long-acting amylin injection MET-233i showed promising weight-loss after a little more than a month of treatment.
Empaveli reduced proteinuria by 68% versus placebo in glomerulopathy and glomerulonephritis, an effect that was sustained through one year of follow-up.
Atopic dermatitis manifests differently in patients with darker skin color, according to the companies, including subtler presentations and more severe and prolonged lesions compared to patients with lighter skin.
As multiple companies vie to expand on Alnylam’s success in commercializing RNAi therapeutics, the pioneering company has set a goal of targeting small interfering RNA to any tissue by 2030.