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FDA grants rare pediatric disease status to AGT-181 for Hurler syndrome
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The FDA has issued rare pediatric disease designation to AGT-181 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, the manufacturer press release said.
AGT-181 (ArmaGen Inc.), an investigational enzyme replacement therapy for the treatment of Hurler syndrome, may help address the neurological complications by targeting certain receptors to allow noninvasive crossing of the blood-brain barrier into the central nervous system.
“Receipt of rare pediatric disease designation strengthens ArmaGen’s commitment to providing children, families and physicians with treatment options for the currently unaddressed complications of Hurler syndrome,” James Callaway, PhD, CEO of ArmaGen Inc., said in a press release.
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.