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Fierce Pharma5d

FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients

Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...
STAT12d

Pharmalittle: We’re reading about Sarepta resisting FDA over Elevidys, agency naming top drug regulator, and more

Sarepta Therapeutics refused a FDA request to halt shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy ...

FDA probes death of patient on Sarepta's Elevidys, partner Roche says death unrelated to therapy

The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received ...
Fierce Pharma5d

FDA launches probe into new Elevidys death as Sarepta, Roche stress gene therapy not at fault

In its own communique issued shortly after the FDA’s, Sarepta elaborated on the situation and confirmed a Roche statement to ...
BioSpace5d

Sarepta Denies Latest Patient Death is Elevidys-Linked as FDA Launches Probe

Brazilian authorities said the death was unlikely to have been caused by Elevidys and was instead more in line with severe ...

FDA’s Reversal on Gene Therapy Helps My Son

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.
Fierce Pharma2d

Vinay Prasad's exit signals FDA's probable slip toward more permissive regulation of cell and gene therapy: analyst

But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...
Just The News4d

FDA allows distribution of muscular dystrophy drug again after public criticism

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...