Mitsubishi Tanabe pulls Radicava app after skeptical EU regulators ask for new trial

When Mitsubishi Tanabe’s Radicava got its nod from the FDA in 2016, it marked a turning point for ALS patients after 20 years without a new treatment on the market. But despite the hype—and with the drug’s eye-popping price tag in mind—Europe’s governing body asked to see more evidence that it really works.

Mitsubishi pulled its EU marketing application for Radicava on Friday after the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) called for an additional 12-month, placebo-controlled study to answer lingering concerns about the drug’s effectiveness.

At issue was the duration and patient pool in a phase 3 study that helped the treatment secure its FDA nod. The CHMP admitted the 24-week study did show “significant improvements” in certain ALS scores over placebo, but said it didn't show Radicava aided breathing, boosted muscle strength or helped patients live longer.

The committee also pointed out that the study was small and brief—only 137 patients and 24 weeks—and that a higher number of those taking Radicava came into the trial with less severe disease. Patients later switched from placebo to Radicava saw “no noticeable effect” afterward, the CHMP noted.

"At the time of the withdrawal, the Committee was of the opinion that, because of lack of proven effectiveness, the benefits of Radicava did not outweigh its risks," the CHMP said on its website.

The company, unsurprisingly, disagrees. “(Mitsubishi Tanabe) believes the efficacy and safety of (Radicava) has been demonstrated and an additional long-term, placebo-controlled study is unwarranted,” the company said in a release.

The CHMP considered offering Radicava a conditional approval, which would allow it to launch now and deliver further data later. Mitsubishi Tanabe offered up a registry study that would compare patients using Radicava to those treated with other agents in the past.

“The Committee considered the merits of such a study but had some objections, including the fact that the treatment for ALS had changed significantly over the past few years, rendering comparisons difficult,” the committee wrote.

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Radicava, approved to treat both ALS and acute ischemic stroke in Japan and South Korea before to its U.S. nod, is a free radical scavenger that removes oxygen-containing molecules that build up in people with ALS and damage nerves. In the run-up to its FDA approval, ALS groups touted the drug as a game changer in the field after nearly two decades without a new treatment.

In response, U.S. payers backed the drug in droves despite its high price tag. In 2017, Radicava was priced at $1,086 per infusion with a dosing schedule that begins with 14 straight days of treatment. Despite the treatment’s novelty, GlobalData estimated its launch market at just $38 million in the U.S. back in 2013 with an expected dip to around just $16 million per year in 2020 after generic competition hit the market.

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However, those figures significantly undershot Radicava’s actual performance. In 2018, the drug cleared more than $248 million in sales in the U.S.—a 119.5% increase over the previous year, the company said. However, Mitsibushi Tanabe execs forecast the drug slipping from that high to around $202 million in 2019 after the initial patient pool dried up.