(MENAFN- ProactiveInvestors - N.America) Summit Therapeutics (LON:SUMM NASDAQ:SMMT) has received its second drug designation in two days from the US Food and Drug Adminstration for ezutromid, a treatment for Duchenne Muscular Dystrophy (DMD) .
Having been granted fast track status yesterday, ezutromid has today been classified a 'rare pediatric disease'.
"Rare Pediatric Disease designation builds upon the Fast Track and Orphan Drug designations which the FDA has already awarded to ezutromid," said Glyn Edwards, Summit's chief executive.
"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, he added.
Through the rare pediatric disease designation, a sponsor of can receive a voucher that enables a priority review for another product.
A priority drug approval review takes six months compared to ten months or longer for a standard approval review.
The voucher can be sold or transferred and earlier this year Gilead acquired one from Knight Therapeutics for US$125mln though they have been sold for as much as US$350mln.
The Priority Review Voucher is requested at the time of the marketing application and awarded upon approval of the product.
Summit concedes it may not get a voucher as the programme is set to expire next month although itnotes the possibility it may be extended again by US politicians.
DMD is a fatal disease that affects around 50,000 boys and young men globally and is caused by faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles.
Summit believes that as an utrophin modulator, ezutromid aid all patients with DMD, regardless of their underlying dystrophin gene mutation.
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