FDA approves oral form for the treatment of adults with amyotrophic lateral sclerosis

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FDA has approved Radicava ORS (edaravone) oral suspension for the treatment of adults with amyotrophic lateral sclerosis. Radicava ORS is an orally administered version of Radicava, which was originally approved in 2017 as an intravenous infusion to treat amyotrophic lateral sclerosis.

Radicava ORS is self-administered and can be taken at home. After fasting overnight, Radicava ORS should be taken in the morning orally or through a feeding tube. The oral medication has the same dosing regimen as Radicava—an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period and subsequent treatment cycles consisting of daily dosing for 10 out of 14-day periods, followed by 14-day drug-free periods.

Disease or Condition

Amyotrophic lateral sclerosis is a rare disease that attacks and kills the nerve cells that control voluntary muscles. Voluntary muscles produce movements such as chewing, walking, breathing, and talking. Amyotrophic lateral sclerosis causes the nerves to lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis. amyotrophic lateral sclerosis is a progressive disease that continues to get worse over time. Most cases will result in death from respiratory failure, usually within 3 to 5 years from when the symptoms first appear. According to the latest data from the Centers for Disease Control and Prevention, at least 16,000 Americans have amyotrophic lateral sclerosis.

Effectiveness

The effectiveness of Radicava ORS is based on a study that showed comparable levels of Radicava ORS in the bloodstream to the levels from the IV formulation of Radicava. The efficacy of Radicava for the treatment of amyotrophic lateral sclerosis was previously demonstrated in a six-month clinical trial that served as the basis for approval in 2017. In that trial, 137 participants were randomized to receive Radicava or placebo. At week 24, individuals receiving Radicava declined less on a clinical assessment of daily functioning compared to those receiving placebo.

Safety Information

The most common side effects of Radicava are bruising (contusions), problems walking (gait disturbances), and headaches. Fatigue is also a possible side effect of Radicava ORS. Radicava and Radicava ORS can have serious side effects associated with allergic reactions including hives, rash, and shortness of breath. For patients with sulfite sensitivity, sodium bisulfite—an ingredient in Radicava and Radicava ORS—could cause a type of allergic reaction that can be life-threatening.

The prescribing information includes additional information on risks associated with Radicava ORS.

Designations

FDA granted Radicava ORS Orphan Drug approval, Priority Review, and Fast Track designations.

Source: News Release
US Food and Drug Association
May 12, 2022