News

Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy
Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...
STAT11h
Replimune skin cancer therapy rejected by FDA, a sign of regulators’ hardened stance
The FDA on Tuesday rejected a skin therapy from Replimune Group, suggesting a hardened stance on drug approvals under new agency officials ...
BioSpace12h
Lack of Transparency Tarnishes Sarepta’s Sheen as Patient Deaths Trigger FDA Battle
Sarepta Therapeutics’ stock has dropped precipitously as questions swirl around the safety of its gene therapies. Meanwhile, the Duchenne patient community fears losing access to Elevidys while the ...
STAT13h
Pharmalittle: We’re reading about Sarepta conceding to the FDA, the U.S. transplant system, and more
Sarepta Therapeutics reversed course and agreed to halt all shipments of Elevidys, its gene therapy for Duchenne muscular ...
FDA Rejects Replimune’s Skin Cancer Treatment, Shares Sink
US regulators rejected a skin cancer treatment from Replimune Group Inc. in another sign of the agency’s new leadership ...
Sarepta to pause Elevidys gene therapy shipments in US
Sarepta Therapeutics said on Monday it will pause all shipments of Elevidys in the United States after two teenage boys with a rare condition called Duchenne muscular dystrophy, who had received the ...
MedPage Today1d
Drugmaker Refuses FDA's Request to Pull Gene Therapy Tied to Patient Deaths
The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...
BioPharma Dive1d
5 questions on Sarepta, the FDA and a Duchenne gene therapy crisis
A standoff over Elevidys could have major consequences for Duchenne patients, gene therapy companies and the perception of ...
MPR1d
Third Death Prompts FDA Action on Sarepta’s Gene Therapy Products
The third death occurred in an adult male with limb girdle muscular dystrophy who received a Sarepta AAVrh74 gene therapy product, SRP-9004, in a phase 4 clinical trial.
Fierce Biotech1d
Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy
The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb girdle ...
Precision Medicine Online1d
Sarepta to Continue to Ship Duchenne Gene Therapy Elevidys Despite FDA Request
The FDA requested Sarepta voluntarily stop distributing Elevidys after the death of a third patient who received one of the firm's gene therapies.
Cambridge pharma company lays off almost 500 amid FDA dispute over treatment
A Cambridge-based company developing gene therapies for rare diseases is laying off more than a third of its workforce.