JUPITER, Fla., Aug. 22, 2017 /PRNewswire/ -- Jupiter Orphan Therapeutics, Inc. ("JOT"), Jupiter, FL, today announced that it has received notification from the US Food and Drug Administration (FDA) that its Orphan Drug Designation request for trans-Resveratrol has been granted.
"Orphan Drug Designation serves as an important milestone for JOT as it positions our JOTROL™ product as a potential treatment for FA. We are hopeful that JOTROL™ can ultimately provide a meaningful treatment for FA patients around the world based on the earlier Phase II trial, utilizing resveratrol, conducted by our partner Murdoch Children's Research Institute, Melbourne, Australia ("MCRI"). That trial was an open label trial and generated very encouraging results, where 4 out of 5 very important endpoints were met. We, JOT together with MCRI, expect to repeat these results through a larger placebo controlled study utilizing JOTROL™ to avoid the Gastro Intestinal (GI) tolerability issues. This will hopefully lead to market approval in several territories, including USA" said Chief Executive Officer, Christer Rosén of JOT.
JOT has developed a unique patented formulation of trans-resveratrol called JOTROL™. JOTROL™ is expected to deliver the well documented high amount of resveratrol in blood plasma that is required to achieve therapeutic effects. These high doses have earlier been plagued with severe GI-side effects that has stopped utilization of resveratrol in the pharmaceutical field. JOT is expecting, based on very successful pre-clinical data, that resveratrol administration in the JOTROL™ formulation will deliver the necessary levels of resveratrol in plasma without generating any severe GI side-effects.
About Friedreich's Ataxia (FA)
Friedreich's Ataxia (FA) is a rare inherited disease that causes damage to the nervous system as well as diminished mobility. FA usually begins in childhood and leads to impaired muscle coordination (ataxia) which worsens over time. It is caused by a defect (mutation) in a gene Frataxin (FXN). Friedreich's ataxia is recessive, meaning it only occurs in someone who inherits two defective copies of the gene, one from each parent. Although rare, FA in the most common form of hereditary ataxia, affecting about 1 in 50,000 people in the United States. There are no approved treatments available today. Visit the FARA (Friedreich's Ataxia Research Alliance) website, www.curefa.org, for further details on this rare disease.
About Jupiter Orphan Therapeutics
Jupiter Orphan Therapeutics, Inc. (JOT) is a clinical stage specialty pharmaceutical company developing therapies for rare diseases. JOT, a Delaware Corporation with its principal office located in Jupiter, FL, USA, was founded in the summer of 2015. In its short period of operations, JOT has assembled a very strong management and scientific team, developed JOTROL™ as a platform product to treat multiple rare diseases as well as collaborating with established partners in other disease areas.