FDA Grants IntraBio Pediatric Disease Designation for Experimental Niemann-Pick Disease Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to IntraBio’s  lead compound series (IB1000s) for the treatment of Niemann-Pick disease Type C (NPC), a rare, debilitating, inherited lysosomal storage disorder.

NPC is caused by dysfunction of the NPC1 protein leading to the accumulation of lipids in lysosomes, resulting in impaired cell function and cell death in various organs. The disease typically begins in early childhood and is chronic and progressive in nature. Motor and cognitive symptoms become more disabling as the disease progresses. Currently, the average age of death for NPC patients is approximately 10 years, with half of the patients dying before the age of 12.5 years.

IB1000s are a series of orally administered, modified amino-acid analogs/esters. IB1000s are believed to have an effect on the normalization of neuronal membrane potential and intracellular ion regulation via calcium channels and have been observed to have symptomatic and neuroprotective properties and disease modifying potential.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes IB1000s eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. IntraBio has previously been granted Orphan Medicinal Drug designation from the European Commission and Orphan Drug designation from the FDA for IB1000s for the treatment of NPC.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Ultragenyx and Kyowa Hakko Kirin sold their Priority Review voucher for $80.6 million.

“The Rare Pediatric Disease designation for IB1000s emphasizes the need for the development and commercialization of rare-disease drugs,” said Mallory Factor, chairman and CEO of IntraBio. “The designation is an important step in enabling us to develop novel therapeutic options in these areas of high unmet medical needs.”

December 3, 2018
Photo: Mallory Factor, CEO of IntraBio