Summit Receives Fast Track Designation from FDA for Ezutromid in the Treatment of Duchenne

Summit Therapeutics plc today announced it has received Fast Track designation from the US Food and Drug Administration (FDA) for ezutromid in the treatment of Duchenne. Ezutromid is a utrophin modulator, which has the potential to treat people living with Duchenne regardless of their specific gene mutation. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions that address an unmet medical need. We look forward to ezutromid's continued progress in its ongoing Phase 2 clinical trial, PhaseOut DMD.

Read Summit's Press Release:

Oxford, UK, 26 September 2016 - Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, today announces it has received Fast Track designation from the US Food and Drug Administration ('FDA') for ezutromid in the treatment of DMD. Ezutromid is a utrophin modulator and represents a potential disease modifying treatment for all patients with the fatal muscle wasting disease DMD.

"Fast Track designation underscores the importance that the FDA places on developing new treatments for life-threatening disorders, such as DMD, and aligns well with our recently outlined strategy to accelerate the development of ezutromid to market," said Glyn Edwards, Chief Executive Officer of Summit. "As a utrophin modulator, ezutromid has the potential to significantly advance the state of care for all patients with DMD, regardless of their underlying genetic fault.  We look forward to ezutromid's continued progress in our ongoing Phase 2 clinical trial, PhaseOut DMD."

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions that address an unmet medical need. Advantages of Fast Track designation include opportunities for more frequent interactions with the FDA during all aspects of development, submission of a New Drug Application ('NDA') on a rolling basis, and eligibility for accelerated approval and priority review. This designation is in addition to ezutromid being granted Orphan Drug designation by the FDA and the European Medicines Agency.

About Utrophin Modulation in DMD

DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties. Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin has a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company's lead utrophin modulator, ezutromid, is an orally administered, small molecule. DMD is an orphan disease, and the US Food and Drug Administration and the European Medicines Agency have granted Orphan Drug designation to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation by the FDA.

About Summit Therapeutics

Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc).

Views: 1646

Comment

You need to be a member of PPMD Community to add comments!

Join PPMD Community

Comment by Steven on September 26, 2016 at 12:05pm

Given they plan to have 40 + boys enrolled in the phase 2 trial in the next 6 months and some results by Fall 2017, might the FDA, in similar vain to  Eteplirsen approve without the need for a placebo trial?  If it works it would be a game changer as it potentially helps all boys regardless of mutation (and BMD).  

Need help using this community site? Visit Ning's Help Page.

Members

Events

© 2019   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service