Topic: Duchenne muscular dystrophy
Sarepta is hoping to avoid the data-quality controversy that arose over its Duchenne muscular dystrophy drug Exondys51 last year with its next-generation drug golodirsen.
A panel of experts convened by the FDA has overwhelmingly knocked back PTC Therapeutics’ filing for approval of ataluren.
Here's Tuesday's dose of your biopharma news of note.
Shares in the biotech shot up 60% after it posted phase 2 data that suggest it can muscle in on a market fought over by Boehringer and Roche.
Sarepta CEO Doug Ingram aims to raise $250 million in a public stock offering as the company looks beyond its first product, DMD therapy Exondys 51.
Sarepta offers Exondys 51 outside of the U.S. through a managed access program run by Clinigen, as an EMA decision is expected next year.
After three years, Johnson & Johnson is ducking out of a partnership with Capricor focused on the use of stem cells to treat cardiovascular disease.
Bristol-Myers Squibb has offloaded two neurodegenerative disease programs to Biogen and Roche in separate deals.
Akashi Therapeutics has been given the FDA go-ahead to restart tests for its Duchenne muscular dystrophy candidate HT-100 (delayed-release halofuginone), just over a year after the test was suspended after a death.