Sarepta Therapeutics is anticipating full FDA approval and a label expansion for its gene therapy, Elevidys, with the company indicating on its Q1 earnings call last week that a draft label is imminent. But the therapy has come under the microscope regarding its high cost and clinical effectiveness.
Tag Archive for: gene therapy
Pfizer’s investigational Duchenne muscular dystrophy gene therapy fordadistrogene movaparvovec has been hit with another patient death, forcing the pharma to pause dosing in its Phase III study.
A record 8,000 attendees are expected at this week’s 27th Annual Meeting of the American Society of Gene & Cell Therapy in Baltimore to discuss the opportunities and challenges facing the industry. Billed as the largest gathering of gene and cell therapy professionals, ASGCT 2024 starts tomorrow and will run through the end of the week, bringing together stakeholders from therapeutic developers and investors to patient advocates, policymakers and regulators.
The FDA’s authorization of therapies that fail their efficacy endpoints casts doubt on the regulator’s accelerated approval pathway, according to an article from David Rind, chief medical officer of the drug pricing non-profit Institute for Clinical and Economic Review, published in the Journal of the American Medical Association. In his viewpoint piece, Rind specifically called out Sarepta Therapeutics’ gene therapy Elevidys, which won the FDA’s accelerated approval in June 2023—despite falling short of its primary efficacy endpoint in two studies.
Pfizer will go toe-to-toe with CSL Behring following the FDA’s Friday approval of its hemophilia B gene therapy Beqvez and will launch a warranty program based on the durability of response.
In 2023, cell and gene therapy saw an unprecedented surge with seven FDA approvals, and this year, an even greater number of these treatments could reach the market. So far in 2024, the regulator has given the green light to three new CGTs, and at least seven additional cell and gene therapy products are expected to receive approval by year’s end, according to a March report from the Alliance for Regenerative Medicine.
“The FDA Fast Track Designation for AB-1002 is an important accomplishment for the clinical development of this program and highlights our goal of bringing potentially effective treatments to patients with advanced congestive heart failure,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio.
2023 was a banner year for cell and gene therapy, with seven FDA approvals, according to the Alliance for Regenerative Medicine. And the pace doesn’t appear to be slowing at all as the second quarter of this year gets underway. The advocacy group is anticipating an even bigger year for the CGT space in 2024, with a potential 17 approvals in the U.S. and EU combined, according to ARM’s annual Cell & Gene State of the Industry Briefing in January.
The company said it has identified “material weakness in its internal control over financial reporting”, which resulted in ineffective disclosure of controls and procedures.
Orchard Therapeutics, a unit of Japan’s Kyowa Kirin , priced its newly approved gene therapy for a rare, life-threatening genetic disease on Wednesday at $4.25 million, making it the most expensive drug in the United States.
