News

Sarepta's licensing patner Arrowhead expects near-term payments despite setbacks

Arrowhead Pharmaceuticals said on Wednesday it expects to receive near-term milestone payments from Sarepta Therapeutics as ...
BioPharma Dive21h

Replimune drug rejected by FDA; Omega raises $647M biotech fund

Analysts questioned whether the complete response letter Replimune received was evidence of changing approval standards at ...
BioSpace22h

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 ...
STAT1d

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 ...
KFF Health News1d

Sarepta Suspends Elevidys Shipments After Pressure From FDA, Hospitals

Sarepta's pause on its prescription gene therapy drug for Duchenne muscular dystrophy gives the company time to work with the FDA on reviews. Infusions nationwide will be canceled. Also in the 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 ...

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

While Sarepta has now consented to the FDA’s request to stop selling Elevidys, the company’s brief standoff with the agency ...
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.
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.