Replies to post #203150 on Biotech Values

[bladerunner1717]

FDA to consider Illinois-based company's DMD drug

http://www.chicagotribune.com/business/ct-marathon-dystrophy-drug-0811-biz-20160810-story.html

FDA to consider approving Northbrook company's muscular dystrophy drug
Deflazacort
Chemical structure of Deflazacort. (U.S. National Library of Medicine)
Lisa SchenckerContact Reporter
Chicago Tribune
The Food and Drug Administration has agreed to consider green-lighting a drug that could be among the first of its kind approved to treat a childhood-onset form of muscular dystrophy, Northbrook company Marathon Pharmaceuticals announced Wednesday.

Marathon has the U.S. rights to the drug, called deflazacort. The pharmaceutical is already available and used to treat Duchenne muscular dystrophy outside the U.S., where it's approved for other uses. But because the steroid does not have FDA approval, it's not widely available here.

"This would be a big step forward for families across the country to have access to the drug in the U.S.," said Valerie Cwik, Muscular Dystrophy Association executive vice president and chief medical and scientific officer.

Duchenne muscular dystrophy is a genetic disorder that mostly affects boys. It can cause movement, heart and breathing problems and typically leads to death in early adulthood, Cwik said. It's the most common form of childhood-onset muscular dystrophy, she said.


Now, those with Duchenne are often treated with prednisone, also a steroid, though it's not specifically approved to treat the disorder.

Prednisone, however, can cause more weight gain as a side effect than deflazacort, Cwik said.

The FDA agreed to give the deflazacort drug applications — one for tablet and one for a liquid — priority review status, meaning it could have a decision in six months rather than 10. A decision is expected in February, according to Marathon.

One other drug meant to treat the disease, eteplirsen, developed by Sarepta Therapeutics, is also under FDA review.

During the FDA review process Marathon is offering the drug for free to patients in the U.S. who work with participating doctors through its FDA-authorized Access DMD program, said Tim Cunniff, Marathon's executive vice president of research and development. To learn more visit www.AccessDMD.com or call 844-800-4363.

[e-mail]

Twitter @lschencker


Bladerunner

[iwfal]

SRPT PTCT

thought this interesting (can't disagree with them) criticizing FDA for not treating applicants fairly (I think FDA already opened themselves up to this and other criticisms in how they've handled DMD drugs)

Yep. Welcome to the very predictable contagion effects of allowing bad science.

As for recent developments for SRPT - it has now been about 2 months since the SRPT PR that said the FDA wanted WB data from already extant biopsy data. But there has been no PR saying this has happened. And now the recently released 10Q is silent on whether the submission happened. I think it is safe to say that they either do not have valid biopsies they thought they did, or the FDA didn't like the resulting WB analysis (I purposely left out the possibility that SRPT didn't like it because, realistically, SRPT likes any and all data via post hoc 'reasoning' - aka excuses).

BTW as a measure of the extreme disfunction of the DMD activist community, there has been no appreciable comment by them on the FDAs reluctance to accept the NDA for a new DMD drug that actually hit the ITT primary endpoint of a ph3. Yep, wasn't as glamorous (e.g. Fixing the genes), but would allow steroid sparing which would be huge QOL gain. But ... . When both the scientists and the activists in a research area are disfunctional there are meaningful opportunities missed.