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.
For more information, visit http://www.jupiterorphan.com/.
Media contact:
Christer Rosén, +1 561 308-7780
173047@email4pr.com
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SOURCE Jupiter Orphan Therapeutics, Inc.