Fosmetpantotenate is currently being evaluated in a pivotal Phase 3 trial which, if successful, has the potential to be the first approved therapy targeting the underlying cause of pantothenate kinase-associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood.1

Estimated to affect only up to 5,000 people worldwide, this rare genetic condition is characterized by a host of progressively debilitating movement disorders.1,2 There are no approved treatment options for PKAN and current therapeutic strategies are limited to symptom management.3

Fosmetpantotenate is designed to pass the blood-brain barrier, be converted to phosphopantothenic acid (PPA) and restore coenzyme A (CoA) levels. PPA synthesis is a key step in the biosynthesis of CoA, which is essential in biochemical reactions impacting energy metabolism, membrane integrity, signaling and other critical processes.

In December 2018, Retrophin completed patient enrollment in the pivotal Phase 3 FOsmetpantotenate Replacement Therapy (FORT) Study (, which is an international, randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of fosmetpantotenate in approximately 82 patients with PKAN aged six to 65. The FORT Study is designed to be registration-enabling in the U.S. and Europe and top-line results from the study are expected in the third quarter of 2019.

The FORT Study ( is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA). As part of this process, Retrophin and the FDA have agreed that the design of this pivotal trial will be adequate to support a New Drug Application (NDA) if the data from the trial are positive. Detailed information about this pivotal trial can be found at

The FDA and European Commission both granted orphan drug designation to fosmetpantotenate for the treatment of PKAN. In the U.S., this status provides Retrophin with development and regulatory incentives, as well as seven years of marketing exclusivity, if fosmetpantotenate is approved for PKAN. Similarly, fosmetpantotenate is eligible for a single marketing authorization that is valid in all EU countries and 10 years of market exclusivity in the region, upon approval for PKAN. Fosmetpantotenate was also granted fast track status in the U.S., which is designed to facilitate the development and expedite the regulatory review of medicines intended to treat serious conditions with unmet medical needs, in order to reach patients earlier.

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  1. Gregory, et al. GeneReviews. 2002.
  2. Data on file. San Diego, CA: Retrophin, Inc.; July 2016.
  3. NORD Report on PKAN. Pantothenate Kinase-Associated Neurodegeneration. Available at: Last accessed July 26, 2016.