We partner with researchers from around the world to conduct clinical trials studying our investigational therapies.
We continue to enroll patients with pantothenate kinase-associated neurodegeneration (PKAN) in our FOsmetpantotenate Replacement Therapy (FORT) Study which is an international, randomized, double-blind, placebo-controlled study evaluating fosmetpantotenate for the treatment of PKAN, a rare, genetic neurological disorder.1 This Phase 3 clinical trial is designed to evaluate the safety and efficacy of fosmetpantotenate in approximately 82 patients with PKAN aged 6 to 65 years. The first patient was dosed in July 2017; we expect to begin pediatric enrollment in the second quarter of 2018.
The primary endpoint will be the change in score on the Pantothenate Kinase-Associated Neurodegeneration Activities of Daily Living (PKAN-ADL) scale, from baseline through 24 weeks of treatment. After completing the 24-week treatment period, all patients will be eligible to receive RE-024 as part of an open-label extension. The PKAN-ADL is a novel, PKAN-specific, patient-reported outcome scale measuring motor abilities to function in daily living for patients with PKAN. The scale is an adaptation of Part II of the comprehensive and widely-referenced Unified Parkinson's Disease Rating Scale (UPDRS). For the purposes of this trial, the UPDRS was adapted to be optimally relevant to PKAN through a systematic revision involving experts, patient advocacy leaders and regulatory interaction.
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 Agency have agreed that the design of this pivotal trial is will be adequate to support a New Drug Application (NDA) if the data from the trial are positive. 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.
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.
In the second quarter of 2018, we initiated DUPLEX, a pivotal Phase 3 clinical trial of sparsentan in focal segmental glomerulosclerosis (FSGS), which is designed to include an interim analysis of proteinuria to serve as the basis for an NDA filing for Subpart H accelerated approval of sparsentan. The confirmatory endpoint of the study is expected to compare changes from baseline in estimated glomerular filtration rate or, eGFR, which is widely regarded as the best overall measure of kidney function. Sparsentan could be the first FDA-approved pharmacologic treatment option for patients with this rare kidney disorder that often leads to end-stage renal disease.2
In the Phase 2 DUET study, which is currently in its open-label phase, the overall sparsentan treatment group experienced statistically significant reductions in proteinuria, and results showed sparsentan was generally safe and well tolerated in the study.3
- Gregory, et al. GeneReviews. 2002.
- Middleton, et al. Nephrology Rounds 2007; 5(4).
- Trachtman H, Nelson P, Radko K on behalf of the DUET Investigators. Efficacy and Safety of Sparsentan, a Dual Angiotensin II and Endothelin Type A Receptor Antagonist, in Patients with Focal Segmental Glomerulosclerosis: A Phase 2 Trial (DUET). Presented at the American Society of Nephrology Kidney Week Meeting; November 15-20 2016, Chicago, IL.