Tag Archive for: Duchenne muscular distrophy

Pfizer’s investigational Duchenne muscular dystrophy gene therapy, fordadistrogene movaparvovec, failed in a late-stage study to significantly improve motor function in patients versus placebo.

The 2024 American Society of Gene & Cell Therapy annual meeting attracted a record number of attendees and abstracts, testimony to the tremendous growth of the cell and gene therapy industry. But for me, my week onsite in Baltimore laid bare the major challenges that stand between cell and gene therapies (CGTs) and the masses.

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. 

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.

The oral drug, to be sold under the brand name Duvyzat, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD, according to the FDA.

Iovance’s lifileucel for advanced melanoma and Madrigal’s resmetirom for nonalcoholic steatohepatitis are just a couple of the key decisions on the FDA’s docket this quarter.

A patient taking part in an FDA-sanctioned clinical trial assessing a CRISPR-based therapy for a rare form of Duchenne muscular dystrophy (DMD) has died. Few details related to the circumstances of the patient’s death are known at this time, including whether or not he had been dosed with the one-time gene therapy.