|Prosensa CEO Hans Schikan|
Prosensa ($RNA) has begun the FDA submission process for its treatment for Duchenne muscular dystrophy, planning to fully file by year's end with hopes of finally winning approval for a drug that has survived a few brushes with failure.
The drug, drisapersen, is eligible for a piecemeal submission thanks to the agency's fast-track program, under which regulators promise a speedier trip through the review process. If all goes according to plan, the agency could hand down a final decision on the treatment as soon as mid-2015.
That puts Prosensa just about even with Sarepta Therapeutics ($SRPT), its principle rival in DMD, which expects to submit its similar eteplirsen to the FDA by year's end, too.
For Prosensa, while approval is hardly guaranteed, simply getting to this put is no small victory. In Phase III results revealed last year, drisapersen failed to beat out placebo in improving walking distance in boys with the muscle-destroying disease, leading deep-pocketed partner GlaxoSmithKline ($GSK) to wash its hands of the drug and sending Prosensa's shares down as much as 70%.
Months later, however, the company came back with new analysis and extension-study data suggesting administering the drug earlier in the disease and lengthening treatment could delay the progression of DMD, and, after a series of encouraging meetings with the FDA, the biotech got back on track with its regulatory plan.
"The commencement of the NDA submission for drisapersen is the culmination of over 12 years of work focused on making treatment options available for DMD patients globally, to improve the lives of boys with this devastating, debilitating childhood neuromuscular disease," Prosensa CEO Hans Schikan said in a statement. "We have worked closely with patient groups, scientists, and clinicians throughout the development of our portfolio of 6 compounds for treating different DMD patient populations, and we pay tribute to everyone in the DMD community, in particular the boys and their families, who have helped us achieve this moment."
- read the statement