The Impact of the Ice Bucket Challenge on Edaravone’s Approval: A Journey Through ALS Drug Development

Before the Ice Bucket Challenge (IBC) captured the world's attention, edaravone, also known as Radicut in Japan and subsequently Radicava in the US, had already been in trials for Amyotrophic Lateral Sclerosis (ALS). It had been approved for ischemic stroke in Japan since 2001. However, the IBC played a pivotal role in accelerating the drug's development and approval process in the United States.

From Japan to the World Stage

In the summer of 2014, when the Ice Bucket Challenge swept the globe, it also sparked a new wave of interest in ALS research and drug development. The challenge, which had raised over $220 million for ALS research, inspired the ALS Association and the US Food and Drug Administration (FDA) to revisit the landscape of drug development for ALS. At that time, there was little activity in this field, and the IBC catalyzed the FDA to conduct an extensive search for partners working on orphan drugs.

The Japan-Flagged Edaravone Trial

Mitsubishi Pharma was already in the late stages of a Phase III trial in Japan for ALS. The company was wrapping up the final studies to submit to the Pharmaceutical and Medical Devices Agency (PMDA) of Japan. In a move that defied conventional approaches, the FDA requested that Mitsubishi apply for orphan drug status. This unconventional move was granted in May 2015, leading to a quick approval in Japan just a month later.

Overcoming Challenges for Global Approval

The translation and adaptation of the drug dossier from Japanese to English was a significant challenge. Notably, the application was based on a study involving 137 Japanese patients with a six-month follow-up. The FDA recognized the importance of this study but also needed justification for its applicability to Western markets. This highlighted the need for thorough translation and validation efforts to ensure the drug's approval in jurisdictions where it had not previously been tested extensively.

The FDA poured resources into helping Mitsubishi prepare the application. Despite the extensive effort, there were still gaps in the filing, and Mitsubishi was left with numerous post-marketing commitments, including mouse studies on pharmacokinetics (PK) and high-dose safety studies. These additional studies were crucial for the FDA and EMA to ensure the drug's safety and efficacy.

A Global Approval and Beyond

Mitsubishi was able to submit an application to both the FDA and the European Medicines Agency (EMA) in August 2016. The FDA approved the drug ahead of schedule on May 6th, 2017, which was notably faster than expected. However, the EMA also approved the drug without delay, further validating the successful global regulatory process.

The journey of edaravone from Japan to the United States, and its subsequent global approval, demonstrated the power of the Ice Bucket Challenge in accelerating drug development and the critical role of international collaboration in bringing novel treatments to patients.

Conclusion

The Ice Bucket Challenge not only raised funds for ALS research but also played a critical role in expediting the development and approval of edaravone for ALS. This case study highlights the significant impact that public awareness and advocacy can have on healthcare and the pharmaceutical industry.