The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Actimed Therapeutics’ S-oxprenolol (ACM-002) for the treatment of amyotrophic lateral sclerosis (ALS).
ALS (also known as Lou Gehrig’s disease) is a progressive, fatal neuromuscular disease that affects nerve cells in the brain and spinal cord, eventually leading to a loss of muscle control and paralysis. ALS prevents motor neurons sending impulses from the brain and spinal cord to the muscles, causing muscle atrophy and weakness and has a mean survival of 3.5 years. Voluntary muscle action is progressively lost and in late-stage ALS patients may have difficulty communicating, swallowing, moving, and breathing. There is currently no cure for ALS.
Cachexia, a complex wasting disease, may occur in the early stages of ALS and has a significant impact on quality of life, proving terminal in some patients. In pre-clinical ALS models, S-oxprenolol has demonstrated significant benefits including slowing disease progression, improvement in survival, attenuation of muscle loss, motor neuron protection, reduction in loss of body mass and improvements in lean body mass.
Robin Bhattacherjee, Chief Executive Officer of Actimed Therapeutics, said: “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. 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 have a potential role in meeting some of the important needs of this under-served patient population.”
FDA Orphan Drug Designation is granted to investigational therapies which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation provides several benefits to drug developers, including certain development cost benefits in the US, increased FDA interaction and eligibility for seven-year market exclusivity following approval.
