Mitsubishi Tanabe Pharma America, Inc. has announced that the US Food and Drug Administration (FDA) has awarded seven years of Orphan Drug Exclusivity (ODE) to RADICAVA ORS® (edaravone) due to its significant contribution to patient care for individuals grappling with amyotrophic lateral sclerosis (ALS). 

This recognition highlights the FDA's acknowledgment of the oral form of edaravone as a crucial advancement for ALS patients, providing a more accessible treatment option.

The FDA's Orphan Drug program aims to strengthen the development of medications targeting rare diseases affecting fewer than 200,000 individuals in the US RADICAVA ORS, having already received Priority Review and Fast Track designations, gained ODE status for its oral suspension route of administration, offering a clinically superior alternative to the intravenous (IV) administration of previously approved RADICAVA® (edaravone). 

This formulation allows for self-administration, either orally or via a feeding tube, in a portable bottle with an oral dosing syringe, eliminating the need for refrigeration or reconstitution before use. Administered in minutes, RADICAVA ORS is recommended for intake in the morning after overnight fasting under appropriate healthcare provider (HCP) guidance.

Dr. Stanley H. Appel of the Houston Methodist Stanley H. Appel Department of Neurology expressed his enthusiasm, stating, "I'm thrilled at the FDA's recognition of ODE to RADICAVA ORS. This comes at a critical time in the ALS treatment landscape and helps ensure that our patients and HCPs have a treatment option validated for several years, including by the pivotal Phase 3 clinical trial."

The US approval of RADICAVA® (edaravone) was rooted in the pivotal Phase 3 study (Study 19 or MCI186-19), demonstrating a 33 percent slowdown in physical function loss compared to placebo over a 24-week period, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). RADICAVA ORS, offering equivalent efficacy to RADICAVA, boasts a similar safety profile confirmed in clinical trials.

Calaneet Balas, President & CEO of the ALS Association, highlighted the significance of an oral formulation in the ALS community, stating, "We recognize the profound impact that an oral formulation has on the ALS community, in part by offering a less burdensome option for people living with ALS."

With over 14,600 ALS patients treated in the US and prescribed by over 2,300 HCPs, RADICAVA and RADICAVA ORS have amassed substantial support from a plethora of clinical trials, post-hoc analyses, and real-world evidence studies, with findings published in over 40 peer-reviewed articles.