Mitsubishi Tanabe pulls ALS drug from EMA assessment

Mitsubishi Tanabe Pharma has withdrawn its EU application for ALS drug Radicava (edaravone), citing ‘unwarranted’ additional data requirements from the EMA.

The drug has been approved in the US, Japan, South Korea, Canada and Switzerland based on results from a a six month trial in Japan involving 137 people that showed those taking Radicava declined less on a clinical assessment compared with those receiving placebo.

However, the EMA’s Committee for Medicinal Products for Human Use (CHMP) has said than an additional study of survival, lasting at least 12 months, would be required to obtain the data necessary for regulatory approval in the EU.

In a statement Mitsubishi Tanabe said it believes “that the efficacy and safety of edaravone has been demonstrated and an additional long-term study is unwarranted”.

Therefore, the company said it “has made the difficult decision to withdraw the marketing authorisation application for edaravone” and “will now carefully consider the future options for edaravone in the EU”.

The company added that it will “continue to make every effort to deliver edaravone to patients suffering from ALS worldwide by increasing, as far as possible, the number of countries with regulator approvals based on current data”.

Radicava is an intravenous infusion given by a healthcare professional. It is administered with a first treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period.

Subsequent cycles consist of dosing on 10 of 14 days, followed by 14 days drug-free.

Mitsubishi Tanabe is also developing an oral formulation of the drug as an option to supplement the existing IV formulation.

Radicava was the first ALS treatment approved by the FDA in more than 20 years. The Association asked the manufacturer to file its drug upon learning about its use to treat ALS in Japan.

The drug does, however, come with a raised risk of serious side effects such as hives, swelling, or shortness of breath, and potentially life-threatening allergic reactions to sodium bisulfite.

And as ever there is the issue of cost – the annual cost before any discount is more than $145,500.