Ayretrade, Inc. (SUMM) Receives FDA Rare Pediatric Disease for Ezutromid as DMD Treatment

September 27, 2016 7:06 AM EDT
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Summit Therapeutics plc (Nasdaq: SMMT) announces it has received Rare Pediatric Disease designation from the US Food and Drug Administration (‘FDA’) for ezutromid in the treatment of DMD. As a utrophin modulator, ezutromid has potential as a disease-modifying treatment for all patients with the fatal muscle wasting disease DMD, regardless of their underlying dystrophin gene mutation.

“Rare Pediatric Disease designation builds upon the Fast Track and Orphan Drug designations which the FDA has already awarded to ezutromid, recognizing a significant unmet medical need in the treatment of DMD,” said Glyn Edwards, Chief Executive Officer of Summit. “We plan to leverage these regulatory advantages in the continued clinical development of ezutromid, which is currently in a Phase 2 clinical trial called PhaseOut DMD, to bring ezutromid to patients in need as quickly as possible.”

The FDA defines ’Rare Pediatric Disease’ as a disease that primarily affects individuals aged from birth to 18 years and affects fewer than 200,000 persons in the US. Under the Rare Pediatric Disease Priority Review Voucher programme, a sponsor who receives an approval for a drug or biologic for a ‘rare pediatric disease’ may qualify for a voucher which can be redeemed to receive a priority review of a subsequent marketing application for a different product. The Priority Review Voucher is requested at the time of the marketing application and awarded upon approval of the product. The voucher may only be used once, but may be sold or transferred an unlimited number of times. The Rare Pediatric Designation follows on the recently granted Fast Track designation by the FDA for ezutromid. The Rare Pediatric Priority Review Voucher programme is set to expire on 1 October 2016, although the US Congress is currently considering legislation to extend the programme. If the programme is not renewed, the FDA will no longer award pediatric vouchers to otherwise eligible sponsors.

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 and Rare Pediatric Disease designation by the FDA.



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