S-oxprenolol granted orphan drug status by FDA as ALS treatment
Therapy seen to reduce muscle and body mass loss in ALS animal models
S-oxprenolol, an investigational therapy being developed by Actimed Therapeutics to treat muscle wasting in amyotrophic lateral sclerosis (ALS), has been granted orphan drug status in the U.S.
That designation, awarded by the U.S. Food and Drug Administration, is given to therapies intended to treat rare diseases, like ALS, that affect fewer than 200,000 people in the country. Orphan drug status provides certain advantages to drug developers, including financial incentives and increased interaction with the FDA. It also allows for seven years of market exclusivity if an approval is ultimately granted.
“We are pleased to receive orphan drug designation for S-oxprenolol in ALS, where loss of body mass and muscle wasting can have a significant impact on survival,” Robin Bhattacherjee, CEO of Actimed Therapeutics, said in a company press release. Actimed is a U.K.-based clinical-stage pharmaceutical specializing in muscle-wasting disorders.
S-oxprenolol designed to treat muscle-wasting condition cachexia
ALS is a progressive disease that damages motor neurons, the nerve cells in the brain and spinal cord that control voluntary movements. As these cells are damaged and die, patients experience symptoms of muscle weakness and wasting, including difficulties in swallowing, breathing, and speaking.
In the early stages of the disease, some people with ALS also develop cachexia, a complex metabolic syndrome in which patients lose weight and muscle mass despite eating normally. The condition can cause fatigue and further exacerbate muscle weakness, significantly impacting quality of life.
S-oxprenolol, also known as ACM-002, is a therapy candidate initially developed by Actimed for the treatment of cachexia in cancer patients. Studies in ALS animal models showed it also may help treat cachexia and disease-related muscle wasting in people with ALS.
ALS is a devastating orphan disease of high, unmet medical need and the early non-clinical data we have generated for S-oxprenolol in this indication suggest that it could play a role in addressing some of the key needs of this under-served patient population.
The drug was found to protect motor neurons and reduce muscle and body mass loss — which ultimately led to a slowing in disease progression and improved survival in the animal models.
“ALS is a devastating orphan disease of high, unmet medical need and the early non-clinical data we have generated for S-oxprenolol in this indication suggest that it could play a role in addressing some of the key needs of this under-served patient population,” Bhattacherjee said.
The company owns the global rights to S-oxprenolol for the treatment of ALS. It has licensed the rights to develop and commercialize the medication for cancer cachexia and other indications to Faraday Pharmaceuticals.
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