Why A New FDA Approved Drug Could Be A Gamechanger For ALS Patients

While most often seen in middle-aged and older adults between 40 and 70 years old, amyotrophic lateral sclerosis (ALS) can affect anyone at any age, according to Johns Hopkins Medicine. It is characterized by the breakdown of motor neurons in the brain and spinal cord. This impaired communication between neurons and muscles leads to muscle deterioration, which may hinder one's abilities to move and speak. In some cases, the condition can result in paralysis, reports the U.S. Food and Drug Administration (FDA).

With five ALS treatment drugs currently authorized by the FDA, a new drug known as Radicava ORS has now also gained FDA approval (via Healthline). As an oral medication, the drug alleviates challenges often faced by ALS patients being treated with Radicava — the intravenous (IV) version of the drug. Able to be taken at home, patients would no longer have to travel to receive an IV, sometimes required as frequently as once a week, explains neurologist Dr. Santosh Kesari via Healthline.

International expert on ALS research and clinical trials Dr. Jinsy Andrews elaborates on how IV treatments are not always accessible for ALS patients, stating via Healthline, "The disease creates difficulty with speaking and swallowing, breathing, walking and using their arms. They don't need to have the additional burden of maintaining an IV access and all the complications that come with that."

Per the FDA's public news release, Radicava ORS would require on-and-off dosing cycles. Taken daily for the first two weeks of treatment, patients would then go without the drug for the following two weeks. Daily usage would then be reduced to 10 days out of a 14-day period before going off the drug again for another two weeks, as the cycle continues. Making the drug even more accessible is the fact that it can also be taken through a feeding tube.

With Radicava ORS thought to be similar in effectiveness as the Radicava IV treatment, the development of additional oral treatments appears to be in the works by drug manufacturers such as Amylyx, who are currently seeking FDA approval for their ALS drug, AMX0035, as reported by Healthline.