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FDA grants rare pediatric disease status to gene therapy for Hurler's syndrome
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The FDA has issued rare pediatric disease designation to a gene therapy product candidate for the treatment of patients with Hurler syndrome, also known as mucopolysaccharidosis type I, according to the drug’s manufacturer.
A genetic disorder characterized by progressive mental decline, loss of physical function, impaired language development, corneal and retinal damage, carpal tunnel syndrome and restricted joint movement, Hurler syndrome is typically diagnosed following development of symptoms at 9 months of age.
Unlike other forms of mucopolysaccharidoses (MPS) which generally have a later onset and milder symptoms with a slower disease progression, patients with Hurler syndrome exhibit developmental delay by the end of the first year, and usually stop developing between 2 and 4 years.
Due to the fact that Hurler syndrome affects approximately only 3,000 children worldwide, there are no currently approved therapies for this disease.
RGX-111 (RegenXBio Inc.), an investigational gene therapy product for the treatment of Hurler syndrome, may help address the neurological complications by using an adeno-associated virus gene therapy to deliver the deficient a-l-iduronidase gene to the central nervous system.
“The Rare Pediatric Disease Designation builds upon the Orphan Drug Designation granted earlier this year by the FDA to RGX-111 for MPS I, underscoring the therapy’s potential to provide meaningful benefit to children struggling with this severely debilitating disease,” Kenneth T. Mills, president and CEO of RegenXBio, said in a press release. “We plan to file an Investigational New Drug application for RGX-111 for the treatment of MPS I in the first half of 2016, and look forward to working with the FDA to advance this important gene therapy to patients in need.”
The FDA has defined a “rare pediatric disease” as a disease that affects fewer than 200,000 individuals in the U.S., primarily aged from birth to 18 years. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a rare pediatric disease may be eligible for a voucher which can be redeemed to obtain priority review for a subsequent marketing application for a different product.