Ultragenyx’s GTX-102 Receives FDA Breakthrough Therapy Designation for Angelman Syndrome

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the most promising stocks according to Wall Street analysts. On June 27, Ultragenyx Pharmaceutical announced that the US FDA granted Breakthrough Therapy Designation for GTX-102 (apazunersen) as a treatment for Angelman syndrome. The designation expedites the development and review of drugs for serious conditions when preliminary clinical evidence suggests substantial improvement over existing therapies.

The FDA’s decision is supported by positive data from a Phase 1/2 study that involved 74 patients, aged 4 to 17 years, all with a full maternal UBE3A gene deletion. Participants in this study showed consistent developmental gains with rapid, sustained, and continuous improvements across multiple symptom domains when treated for up to 3 years.

A research team in a laboratory peering through microscopes at a biologic product.

Angelman syndrome is a rare, neurogenetic disorder caused by the loss-of-function of the maternally inherited allele of the UBE3A gene. In almost all cases, the maternal UBE3A allele is either missing or mutated, leading to limited or no protein expression. This condition affects an estimated 60,000 people in commercially accessible geographies. It is a lifelong neurodevelopmental disorder characterized by cognitive impairment, motor impairment, balance issues, and debilitating seizures.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is a biopharmaceutical company that identifies, acquires, develops, and commercializes novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific.

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Disclosure: None. This article is originally published at Insider Monkey.