MT Pharma America announced that the US Food and Drug Administration (FDA) has granted approval of Radicava (edaravone), as an intravenous infusion treatment for Amyotrophic Lateral Sclerosis (ALS), a rapidly progressive neurodegenerative disease in which the majority of patients die within two to five years of diagnosis. People given Radicava showed significantly less decline in physical function compared to placebo as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument for monitoring the progression of disability in patients with ALS.

“We believe Radicava offers new hope for people with ALS and exemplifies MT Pharma America’s commitment to innovative therapies for patients in the United States battling life-threatening diseases,” said Atsushi Fujimoto, president, MT Pharma America. “We recognize how important this therapy may be to people with ALS and are committed to helping provide access to this important treatment option, with the goal of keeping out-of-pocket costs at a minimum for eligible patients.”

An estimated 5,000-6,000 Americans are diagnosed each year with ALS, an incurable disease that affects the nerve cells in the brain and spinal cord.3,6,7 Initial symptoms can be subtle at first, and it can take 12 to 14 months to be accurately diagnosed with ALS.

“For people with ALS and their families, having a new therapy which slows the decline of physical ability is incredibly significant,” said Jonathan S. Katz, M.D., ALS Clinic director, Forbes Norris MDA/ALS Research and Treatment Center at California Pacific Medical Center. “The approval of Radicava brings us into a new era of treatment by evolving how we manage this complex disease. This is an uplifting milestone for the ALS community especially since it’s been so long since we had anything new.”

The comprehensive clinical development program for Radicava in ALS spanned 13 years and included multiple phase 3 trials. The pivotal phase 3 study (MCI186-19), which evaluated 137 people with ALS, formed the basis for the FDA approval of Radicava. Data demonstrated patients who received Radicava for six months experienced significantly less decline in physical function — by 33 per cent or 2.49 ALSFRS-R points (p=0.0013).

The most common adverse reactions that occurred in greater than 10 percent of patients and greater than placebo were bruising (contusion), problems walking (gait disturbance) and headache.

MT Pharma America is helping to make Radicava accessible to all patients who need it and has created Searchlight Support, a patient access program for people with ALS who are prescribed Radicava. As soon as a person receives a prescription, he or she can access Searchlight Support, including personal case management, reimbursement support and 24/7 clinical support. Some people with ALS who have received prescriptions for Radicava may be eligible to receive additional assistance from MT Pharma America.

“This is an important time for people living with ALS. The approval of RADICAVA gives great promise for what we hope will be the first of many new treatments,” said Barbara Newhouse, President and CEO, ALS Association. “We applaud the work MT Pharma America and the FDA are doing as they have taken unprecedented steps to get this treatment into the hands of patients as quickly as possible.”

Study MCI186-19 was a pivotal phase 3 study that evaluated the efficacy and safety of Radicava compared with placebo in 137 people with ALS. In the study, after a 12-week pre-observation period, eligible patients were randomized 1:1 to receive Radicava 60 mg intravenously for 60 minutes or placebo during a six-month double-blind placebo-controlled phase. The primary endpoint for the study was change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score from baseline to six months.

Radicava is administered in 28-day cycles by intravenous infusion. It takes 60 minutes to receive each 60 mg dose. For the initial cycle, the treatment is infused daily for 14 consecutive days, followed by a two-week drug-free period. All cycles thereafter are infused daily for 10 days within a 14-day period, followed by a two-week drug-free period.

Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and will be commercialized in the US by MT Pharma America. MTPC group companies began researching ALS in 2001 through a comprehensive clinical platform over a 13-year period. In 2015, edaravone was approved for use as a treatment for ALS in Japan and South Korea.

MT Pharma America is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation’s (MTPC) 100 percent owned US holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. MTPA is dedicated to delivering innovative products that address the unmet medical needs of patients in the US.