First results from FORT study for PKAN

The pharmaceutical company Retrophin had developed the drug fosmetpantotenate for the treatment of PKAN. This was tested for safety and efficacy as part of a placebo-controlled and multicenter double-blind clinical trial (FORT trial). This study was completed in July. An extension study, in which all study participants receive the drug, has not yet been completed.

On August 22, Retrophin presented the first results from the FORT study at a Press release (in English).

Prof. Dr. Thomas Klopstock, medical director of the study, has developed a Statement published.

It is disappointing for all PKAN patients and their families that the study was not able to produce clearly positive results as hoped. However, detailed analyses are still pending.