Fosmetpantotenate is an investigational therapy being developed for the treatment of pantothenate kinase-associated neurodegeneration (PKAN) , a devastating neurological disease. The investigational candidate is currently in a pivotal Phase 3 clinical trial. The FDA has granted Fast Track status to fosmetpantotenate, allowing for expedited regulatory review. It also has gained orphan drug designation from the FDA and the European Medicines Agency.


PKAN is a rare genetic condition estimated to affect up to 5,000 people worldwide. In many cases, the first symptoms of PKAN can be seen in early childhood. PKAN is the most common type of neurodegeneration with brain iron accumulation (NBIA). Symptoms include dystonia (sustained muscle contractions causing repetitive movements), muscular rigidity, cognitive impairment, and difficulty speaking. No FDA-approved treatment options exist for PKAN; current options only treat symptoms. Learn more about PKAN .


At the root of PKAN are mutations in the PANK2 gene, which cause a reduction in the activity of the PanK2 enzyme. In the normal biochemical process, the PanK2 enzyme converts pantothenate (PA, vitamin B5) to phosphopantothenate (PPA), and is required for the production of coenzyme A (CoA). CoA is essential for many critical biochemical reactions in the body, including energy metabolism, membrane integrity and cell signaling. Fosmetpantotenate is a novel investigational small molecule replacement therapy designed to pass the blood-brain barrier and be converted to PPA. Our investigational approach is to attempt to restore CoA levels with fosmetpantotenate.


Retrophin has completed enrollment of an international, randomized, double-blind, placebo-controlled study evaluating fosmetpantotenate for the potential treatment of PKAN. This Phase 3 clinical trial, known as FORT (FOsmetpantotenate Replacement Therapy), is designed to evaluate the safety and efficacy of fosmetpantotenate in approximately 82 patients aged 6 to 65 years. The primary outcomes are the change in score of a PKAN-specific activities of daily living (PKAN-ADL) measure, and the safety and tolerability by occurrence of adverse events, and safety assessments. Learn more .

The FORT Study is being conducted under a special protocol assessment (SPA) agreement with the FDA. As part of this process, Retrophin and the FDA have agreed that the design of this pivotal trial may adequately support a New Drug Application (NDA) if the data from the trial are positive.