Pantothenate kinase-associated neurodegeneration (PKAN) is a neurodegenerative disorder caused by a mutated PANK2 gene. This mutation disrupts a metabolic pathway, decreasing the body’s levels of CoA. CoA, or coenzyme A, is an essential component of critical processes such as energy metabolism, signaling, and membrane integrity. The condition can be life-threatening and can cause dysphagia, dystonia, visual impairment, and rigidity as well as other serious complications.
Retrophin Inc. has been working on a Phase 3 study for PKAN and they’ve just announced that enrollment for the trial is complete. That means that we should hear results from the study by the third quarter of next year.
Retrophin’s Phase 3 PKAN trial is called the FORT study. It is examining the efficacy and safety of fosmetpantotenate for patients age 6 to 65 with the condition. 82 participants are enrolled in total and the trial is international. It will evaluate each patients change on the PKAN-ADL (Activities of Daily Living) scale from baseline.
The trial will last for 24 weeks. After the trial is complete, all participants will still have access to the drug through an open-label extension.
The FDA has confirmed through a Special Protocol Assessment (SPA), that the trials design supports the filing of a New Drug Application for fosmetpantotenate.
Fosmetpantotenate has already been given Orphan Drug Designation and Fast Track Status from the FDA. Its Phase 1 study showed the drugs tolerability and preclinical analysis showed its efficacy.
If approved by the FDA, this drug could be the very first approved treatment which addresses the underlying cause of PKAN. Keep your fingers crossed for positive trial results in 2019!
You can read more about this new Phase 3 trial here.