Independent Data Monitoring Committee Clears Way for Retrophin to Enroll Pediatric Patients in PKAN Study

June 4, 2018

Rare Daily Staff

Retrophin said that the Independent Data Monitoring Committee for its pivotal late-stage FORT study for its experimental drug fosmetpantotenate for the treatment of pantothenate kinase-associated neurodegeneration, a rare and life-threatening neurological condition, has completed its scheduled clinical safety review data in adult patients and cleared the way for the initiation of enrollment in pediatric patients aged 6 to 17.

PKAN is a disorder characterized by a host of progressively debilitating symptoms that typically begin in early childhood. People suffering from PKAN may experience movement disorders, such as dystonia, rigidity, problems swallowing, and twisting and writhing, as well as visual impairment. PKAN is estimated to affect up to 5,000 people worldwide.

PKAN is caused by a mutation in the PANK2 gene, which encodes a critical protein that phosphorylates vitamin B5, generating phosphopantothenate. The disruption of this metabolic pathway ultimately leads to decreased levels of Coenzyme A. Fosmetpantotenate is a novel, investigational, small molecule replacement therapy that aims to restore levels of Coenzyme A, a naturally occurring molecule that is essential in many cellular functions and is decreased in individuals with PKAN.

The FORT study is a pivotal phase 3 clinical trial conducted under a Special Protocol Assessment agreement with the U.S. Food and Drug Administration. Under the terms of the SPA agreement, the FORT study protocol required an initial group of adult patients be treated with fosmetpantotenate to allow a safety assessment by the DMC prior to opening enrollment to pediatric patients aged 6 to 17. The company anticipates completion of patient enrollment around year-end 2018, and top-line data to become available in the second half of 2019.

“The DMC’s review is encouraging for the overall safety and tolerability of fosmetpantotenate and supports our optimism that the FORT Study should ultimately enable us to deliver the first approved and transformative treatment for the PKAN community,” said Bill Rote, senior vice president and head of research and development for Retrophin.

June 4, 2018
Photo: Bill Rote, senior vice president and head of research and development for Retrophin

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