FDA Approves New Oral Therapy to Delay Functional Decline of ALS by 33%

On the 14th, Mitsubishi Tanabe Pharma America (MTPA) announced that the U.S. FDA has approved the marketing of oral edaravone (edaravone, English brand name Radicava ORS) for the treatment of amyotrophic lateral sclerosis (ALS). This is a rapidly progressive neurodegenerative disease that currently has no cure. In a pivotal clinical trial, Radicava ORS demonstrated the same efficacy in slowing physical decline compared to an FDA-approved IV formulation. According to the press release, the oral formulation provides ALS patients with a flexible way of administering medication, either by mouth or through a feeding tube, and without the need for dose adjustments.

ALS is a neurodegenerative disease in which muscle function is lost due to the death of motor neurons in the brain and spine. Patients have an average life expectancy of 2 to 5 years after diagnosis, and treatment options are currently lacking.

The intravenous dosage form of edaravone was approved by the US FDA in 2017. Its mechanism of action is still not fully elucidated and is thought to delay disease progression by reducing oxidative stress. Radicava ORS is an oral formulation made for ALS patients. It is a 5 mL liquid formulation that can be taken orally using a syringe or infused into a feeding tube, and does not require refrigeration or be dissolved prior to use. With proper instruction, medical staff can administer the drug in just a few minutes.

The FDA approval is supported by multiple studies, including a pivotal Phase 3 clinical study evaluating 137 ALS patients. Trial results showed that after 24 weeks of treatment, Radicava ORS slowed the decline of physical function in patients by 33% compared with placebo.