The U.S. Food and Drug Administration (FDA) has approved an oral form of edaravone (Radicava ORS), for the treatment of amyotrophic lateral sclerosis (ALS), a rapid neurodegenerative disease. An intravenous (IV) formulation of edaravone was approved in 2017.
ALS, also known as Lou Gehrig’s disease, is a fatal neurodegenerative disease, characterized by the loss of motor neurons in the central nervous system, leading to paralysis and early death.
The oral formulation provides a flexible administration option (taken orally or via feeding tube) and no need for patients to refrigerate or reconstitute the medication before taking.
In a news release, Tulio Bertorini, MD, Professor of Neurology at the University of Tennessee Health Science Center stated, “ALS is a progressive disease that, due to its heterogenous nature, impacts patients at different rates with varying symptoms,” adding, “Therefore, it is crucial that patients have treatment and formulation options that accommodate their own unique needs, and Radicava ORS provides HCPs who have prescribed their ALS patients edaravone with an alternate delivery option.”
The approval of the oral formulation was largely based on multiple clinical trials, including data from the pivotal Phase 3 clinical trial (MCI186-19) evaluating 137 ALS patients that showed treatment with Radicava slowed the loss of physical function by 33% compared to placebo at 24 weeks, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument for monitoring disease progression in patients.
The most common adverse events that occurred in greater than 10% of patients were bruising (contusion), gait disturbance, and headache.
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