First patients in Retrophin trial to receive drug targeting PKAN

October, 2017

In July, the first patients taking part in an international clinical trial on a possible treatment for PKAN, the most common NBIA disorder, received Retrophin Inc.’s drug, fosmetpantotenate, also known as RE-024.

This long-awaited launch of Phase 3 of the trial, which Retrophin delayed until manufacturing issues were resolved, will assess the safety and effectiveness of RE-024. If the San Diego-based company is successful, RE-024 would be the first medication targeting the underlying cause of PKAN, or Pantothenate Kinase-Associated Neurodegeneration. It could change the course of the disease.

People with PKAN cannot make enough of an essential enzyme, pantothenate kinase 2 (PANK2). This enzyme is needed to turn vitamin B5 into an important substance involved in metabolism called Coenzyme A, a lack of which is believed to cause PKAN symptoms. The drug delivers phosphopantothenate to cells, which could help restore the PANK2 enzyme, and, in turn, boost Coenzyme A levels.

RE-024 has been granted orphan drug designation by the U.S. Food and Drug Administration and the European Commission, as well as expedited “fast track” status in the U.S. to facilitate its development and the review process.

The study is being called Fosmetpantotenate Replacement Therapy, or FORT. It follows the gold-standard for a clinical trial, which means that neither the patients nor the doctors know who is getting the drug and who is getting a placebo, or sugar pill.

Retrophin plans to enroll 82 people diagnosed with PKAN between the ages of 6 and 65. Participants must not need a ventilator, cannot have had a deep brain stimulation device implanted within six months and must not have taken deferiprone for 30 days.

After the initial six months of testing, all study participants will have the chance to take the drug.

Right now, four North America study sites are open for enrollment: Massachusetts General Hospital in Boston, Emory University in Decatur, Ga., Children’s Hospital of Pittsburgh, and Rush Movement Disorders Group in Chicago. Dr. Aleksandar Videnovic, from Massachusetts General Hospital, is the lead principal investigator for the U.S. sites, which will be expanded to six or eight, including one on the west coast.

The European arm of the study will enroll patients at multiple sites in Europe this fall and will be led by Dr. Thomas Klopstock at the University of Munich, Germany. He also was the lead investigator for the TIRCON deferiprone study, which has been completed. Final results are being analyzed.

For more information on the FORT study, go to or visit the study’s website at




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