Independent Data Monitoring Committee’s (DMC) review of available safety data supports continuing with study as planned; FORT Study now open to pediatric patients aged 6 to 17
Top-line data anticipated in the second half of 2019
“PKAN is a rare and life-threatening genetic disorder that typically begins in early childhood, so we are particularly pleased to reach this important milestone and begin pediatric enrollment in the FORT Study,” said
Fosmetpantotenate is a novel, investigational, small molecule replacement therapy that aims to restore levels of Coenzyme A (CoA), 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 (SPA) agreement with the
PKAN is a rare, genetic and life-threatening neurological 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 (sustained muscle contraction leading to abnormal posture), rigidity, dysphagia (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 (pantothenate), generating phosphopantothenate. The disruption of this metabolic pathway ultimately leads to decreased levels of CoA.
Forward Looking Statements
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Vice President, Investor Relations & Corporate Communications
Source: Retrophin, Inc.