The Results of This Phase 3 PKAN Clinical Trial are a Major Letdown

According to a story from BioSpace, the biopharmaceutical company Retrophin, Inc., recently released the topline results from its phase 3 clinical trial. This clinical trial was testing the company’s drug fosmetpantotenate as a treatment for pantothenate kinase-associated neurodegeneration (PKAN). While the drug was generally found to be safe and well tolerated during the study, this is the only good news from the trial. Fosmetpantotenate failed to distinguish itself in comparison to placebo and did not satisfy the study’s primary or secondary endpoints.

About Pantothenate Kinase-Associated Neurodegeneration (PKAN)

Pantothenate kinase-associated neurodegeneration (PKAN), which is also known as Hallervorden-Spatz syndrome, is a rare, progressive, degenerative disease that affects brain tissue. The symptoms appear alongside excessive iron accumulation in the brain. PKAN is linked to a mutation affecting the PANK2 gene. The symptoms of PKAN typically begin in childhood and can include retinitis pigmentosa, dystonia, toe walking, difficulty speaking and swallowing, seizures, weakness, limb stiffness, spasms, tremors, and dementia. Most patients lose their mobility within a few years and death by early adulthood is common; infections are the most frequent cause. Treatment options for PKAN are currently severely limited and there is a dire need for more effective options that can slow or halt the progression of the illness. It is estimated that prevalence of the disease is 1 to 3 of every 1,000,000 people. To learn more about PKAN, click here.

About The Trial

The results of this trial are disappointing to say the least, as there is a dire need for more effective treatments for PKAN. This is one of many rare diseases that currently lacks an FDA approved treatment. The study included a total of 84 PKAN patients who were given either fosmetpantotenate or a placebo at a 1:1 ratio. The treatment period of this clinical trial lasted 24 weeks.

While many of the trials that we discuss on our site often have positive results, the reality is that the majority of experimental drugs falter during testing and never make it out of clinical trials. The results of this study are far from the exception. Hopefully, future research will results in the development of a more effective treatment for this deadly rare disease.


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