Drug watchdog criticizes Sarepta gene therapy approval, cost

Alnylam Pharmaceuticals Inc.'s rare disease drug Onpattro has been approved by the Food and Drug Administration, marking the first approval of a therapy that employs the RNA interference (RNAi) gene-silencing technique. Onpattro, also called patis.....»»

Alnylam Pharmaceuticals Inc.'s rare disease drug Onpattro has been approved by the Food and Drug Administration, marking the first approval of a therapy that employs the RNA interference (RNAi) gene-silencing technique. Onpattro, also called patis.....»»

Roche Holding AG made its second major gene therapy deal in a year on Monday, spending $1.15 billion to obtain the rights to Sarepta Therapeutics Inc's investigational drug to treat duchenne muscular dystrophy (DMD) outside the United States......»»

Sarepta Therapeutics Inc (NASDAQ: SRPT)'s new drug application for its second Duchenne muscular dystrophy drug received.....»»

Sarepta Therapeutics Inc. shares fell Thursday after the gene therapy company said an adverse event report was sent to the Food and Drug Administration in error over a muscular dystrophy treatment. Sarept.....»»

A Novartis AG unit that developed a breakthrough gene therapy knew about potential manipulation of animal testing data but failed to disclose it to regulators before the treatment was approved, the U.S. Food and Drug Administration said......»»

The sky-high cost of a new gene therapy isn’t the problem. It’s the dozens of drugs that will use it as a benchmark......»»

Swiss drugmaker Novartis received U.S. approval to sell a gene therapy for $2.125 million per single dose, making it the world's most expensive drug......»»

Brainstorm Health Daily: June 19, 2018 Hello, readers--This is Sy. Shares of biotech Sarepta Therapeutics shot off to the moon Tuesday after the company announced early data on its experimental gene therapy to treat .....»»

Shares of Sarepta Therapeutics Inc. rose 3% in premarket trade Wednesday after the company announced it would acquire biotech Myonexus Therapeutics and its five gene-therapy drug candidates for the.....»»

Pfizer's huge facility in Sanford produces its first FDA-approved gene therapy, which runs $3.5 million a dose......»»

United Therapeutics (NASDAQ: UTHR) has received another Food and Drug Administration approval for Tyvaso more than a decade after the agency first approved the therapy for treatment of pulmonary arterial hypertension.  The FDA approved a supplem.....»»

Here's a roundup of top developments in the biotech space over the last 24 hours. Scaling The Peaks (Biotech Stocks Hitting 52-week Highs Feb. 16) read more.....»»

Here's a roundup of top developments in the biotech space over the last 24 hours. Scaling The Peaks (Biotech Stocks Hitting 52-week Highs Jan. 7) read more.....»»

See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»

See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»

See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»

See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»

See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»

– Expansion of gene therapy collaboration with University of Pennsylvania's Gene Therapy Program extends collaboration by three additional years and adds five new programs – – Orphan Drug Designation recently granted by FDA.....»»