Elysium Health’s EH301 Designated Orphan Drug by FDA for Treatment of ALS

EH301, Elysium Health‘s investigational therapy for amyotrophic lateral sclerosis (ALS), was recently granted orphan drug status by the U.S. Food and Drug Administration.

Elysium’s application for orphan drug designation included data from a 2017 double-blind, placebo-controlled European pilot study in humans.

To expand on the results of the pilot study, Elysium is planning another placebo-controlled trial together with researchers from the Mayo Clinic to evaluate EH301 in up to 150 adults with ALS. The trial is expected to start later this year.

“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,” Leonard Guarente, chief scientist at Elysium Health, said in a press release.

“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,” he said.

Orphan drug designation does not alter the standard regulatory requirements through adequately designed studies to support FDA approval.

The FDA’s orphan drug designation supports the development of safe and effective treatments, diagnosis, or prevention methods of rare diseases, or disorders that affect fewer than 200,000 individuals in the United States. According to the ALS Association, ALS is a rare neurodegenerative disease estimated to affect more than 20,000 Americans.

“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 Eric Sorenson, MD, vice chairman of the neurology department at the Mayo Clinic.

Elysium’s only product on the market so far is Basis (nicotinamide riboside and pterostilbene, or NRPT), a dietary supplement that the company says is designed to support “well-being at the cellular level.”

In a report published earlier this year by ALSUntangled, a group sponsored by the ALS Association, in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, Basis was evaluated as a possible off-label treatment for people with ALS.

Authors of the review read through several studies and evidence of the potential impact of Basis on ALS and concluded that the product has mechanisms of action that could theoretically be useful in treating the disease. Although it appeared safe in a small, short-term trial conducted by Elysium, its therapeutic effects have not yet been evaluated in ALS patients, and it could not be recommended yet to slow, stop, or reverse the progression of ALS, they said.