RE-024 could be the first approved replacement 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
We are planning to initiate the Phase 3 FORT study of RE-024, and expect to begin dosing patients in mid-2017. Detailed information about this pivotal trial can be found at pkanfortstudy.com. The study will be conducted according to a Special Protocol Assessment (SPA) agreement with the FDA. As part of this process, Retrophin and the agency have agreed that the design of this pivotal trial is adequate to support a New Drug Application.
The FDA and European Commission granted orphan drug designation to RE-024 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 RE-024 is approved for PKAN. Similarly, RE-024 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. RE-024 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.
- Gregory, et al. GeneReviews. 2002.
- Data on file. San Diego, CA: Retrophin, Inc.; July 2016.
- NORD Report on PKAN. Pantothenate Kinase-Associated Neurodegeneration. Available at: http://rarediseases.org/rare-diseases/pantothenate-kinase-associated-neurodegeneration/. Last accessed July 26, 2016.