Mitsubishi Tanabe Pharma America (MTPA) announced that it has resumed patient screening and enrolment for its global Phase III clinical trial to assess the long-term safety and tolerability of its drug candidate MT-1186 (edaravone oral suspension) in subjects with Amyotrophic Lateral Sclerosis (ALS) disease.

Sarah Elsayed, Pharma Analyst at GlobalData, a leading data and analytics company, offers her view on the opportunities and challenges in this market:

“ALS patients are at high risk of developing respiratory complications associated with viral and bacterial infections, thus MTPA stated that extra precautions were implemented in the clinical trial sites adhering to COVID-19 protocols. The study operations had to temporarily shut down in March 2020 due to COVID-19 restrictions which resulted in the delay of its estimated completion date from the previously planned date in June 2021 to November 2021. As such, oral edaravone is now expected to launch in the US market sometime in H1 2023.

“The Phase III oral edaravone open-label study is set to enrol up to 150 patients with ALS across various sites in the US, Canada, Europe, and Japan over a duration of 48 weeks of treatment. Participants will receive 100 mg oral edaravone daily according to the same dosing regimen as the IV formulation of Radicava. The initial treatment cycle is a daily dosing for 14 days, followed by a 14-day drug free period then the subsequent dosing is a daily dosing for ten days out of 14 day periods.

“The oral formulation of MTPA’s brand Radicava (edaravone intravenous [IV] infusion), a free radical scavenger, is among the short list of anticipated pipeline drugs to enter the ALS market space within the next five years. Compared to Radicava, edaravone oral suspension is expected to have better patient compliance, less side effects from the IV administration and slightly less annual cost of therapy (ACOT) as there is no need for hospitalization.

“In 2017, Radicava was the first drug to get approved by the FDA for ALS in over 20 years. Radicava generated around $217M global sales in 2019. GlobalData anticipates that the drug will lose patent protection in the US in 2024 and cheap generics will enter the market leading to a decline in the drug’s sales. GlobalData expects that the drug will continue to grow till 2024 with peak sales of $293M globally.

“Currently, Radicava is marketed in the US, Japan and Canada in addition to the gold standard of care for ALS treatment: Sanofi’s Rilutek (riluzole) and its generic versions. Some KOLs interviewed by GlobalData in the US stated that the benefits of Radicava are not significant from their real-life experience and that its long-term benefits do not outweigh the drug’s intense treatment schedule, side effects and high ACOT ($146,883). Further, Mitsubishi had withdrawn the drug’s application to get approved for ALS in Europe as the EMA’s committee for Medicinal Products for Human Use (CHMP) requested further data to provide evidence of Radicava’s effectiveness on larger group of patients. To date Radicava’s future options in the EU are still carefully considered by Mitsubishi.”