NEW YORK, March 29, 2018 /PRNewswire/ -- Elysium Health, Inc.,™ today announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation (ODD) to its experimental therapeutic EH301 for the treatment of amyotrophic lateral sclerosis (ALS). The FDA's ODD program provides orphan status to drugs intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. It is estimated that there are approximately 15,000-20,000 Americans with ALS.
ALS is a rare neurodegenerative disease that affects nerve cells that control voluntary muscles throughout the body to produce movements including talking, eating, walking, and breathing. ALS is progressive, meaning it gets worse over time. As the nerves lose the ability to control muscles, the muscles become weak and eventually lead to paralysis. Most people with ALS succumb to respiratory failure, usually within three to five years from when symptoms first appear.
The Orphan Drug Designation submission included data from a 2017 double-blind placebo-controlled European pilot study in humans. To expand on the results of the pilot study, Elysium Health expects to initiate a placebo-controlled study in collaboration with Mayo Clinic to evaluate EH301 in up to 150 adults with ALS by the fourth quarter of 2018.
As the Vice Chair of Neurology at Mayo Clinic, Dr. Eric Sorenson specializes in neuromuscular medicine and the treatment of ALS. "ALS is one of the most devastating conditions known today, it is generally swift and severe in its onset and progression with no effective treatment options," said Sorenson.
Elysium Health Chief Scientist Dr. Leonard Guarente added that "There is a great deal of work to be done to address the need for continued research to better understand and to treat all neurodegenerative diseases. We believe that the FDA's granting of Orphan Drug Designation for EH301 for ALS underscores the need for novel treatments for this rare condition."
The granting of ODD to EH301 does not alter the standard regulatory requirement through adequate and well-controlled studies to support FDA approval, and there is no guarantee EH301 will be approved for the treatment of ALS by FDA.
Mayo Clinic has financial interest in Elysium Health. All revenue Mayo Clinic receives will be used to fund its not-for-profit mission in medical research and education.
About Elysium Health™
Elysium Health's™ mission is to solve the biggest challenges in health with science, to help people live healthier, longer. Working directly with the world's leading scientists and clinicians, Elysium Health™ is dedicated to translating advances in aging research and technology into effective, scientifically-sound products that help people manage their health in an actionable way. Learn more at www.elysiumhealth.com.
SOURCE Elysium Health