Max Gelman
Senior Editor
Much of the ALS drug news in recent months has focused on Amylyx Pharmaceuticals and its experimental program reviewed by an FDA advisory committee in March. But on Thursday, the FDA greenlighted a new way to take an already-approved ALS drug that flew under the radar.
US regulators OK’ed an oral formulation of Radicava, first approved to treat ALS back in 2017 as a series of IV infusions, to treat adults with ALS, the agency announced Thursday afternoon. The decision gives patients another option for a universally fatal disease with only one other approved drug.
FDA granted the approval to Mitsubishi Tanabe Pharma America, which submitted for the approval in January and was granted priority review.
FDA advisors vote 6-4 against Amylyx’s ALS drug, further dimming its hope for regulatory success ALS is a devastating disease, with most patients dying between three and five years after the onset of symptoms. New treatments are desperately needed, as Radicava and riluzole, approved in the mid-1990s, remain the only two approved drugs.
Whereas riluzole can be taken orally, Radicava (edaravone) previously needed to be administered through several IV infusions. Patients needed to attend clinics or special ALS sites to receive the hour-long infusions for 14 straight days in the first 28-day cycle, and then in 10 out of 14 days for every cycle thereafter.
Now, patients can take the oral formulation at home themselves, or through a feeding tube. The administration schedule remains the same, the FDA said, but patients must also now fast overnight before taking the oral version.
Radicava was granted its first approval after being able to show a modest benefit in slowing functional decline, using a clinically validated yet increasingly controversial measurement. Mitsubishi Tanabe Pharma earned the new OK thanks to a study showing comparable levels of the drug in patients’ bloodstreams to that seen from the IV formulation.
A simple patient survey revolutionized ALS drug development in the 1990s. Its creator says it may be time for an upgrade Thursday’s decision comes as patients have been ramping up the pressure on regulators in the last few years, arguing the review process is too slow. The patient community notched a win back in 2019 when the FDA updated its guidance for developing new ALS drugs, including a provision advising against denying approved treatments in placebo arms.
More recently, patients have rallied around the Amylyx drug in the hopes of swaying the FDA’s experts. At the Amylyx adcomm earlier this year, roughly 30 patients spoke during the nearly two-hour public comment period, stressing the need for new ALS drug approvals.
It remains to be seen whether regulators will greenlight the Amylyx program, dubbed AMX0035. Though the drug achieved statistical significance in a Phase II study measuring function and appeared to show some survival benefit in a long-term follow-up, outstanding questions remained over the quality of the latter dataset.
The FDA has been more open to reviewing the Amylyx drug, however. After initially requesting the biotech conduct a large-scale Phase III trial before submitting its NDA, regulators changed their minds and allowed the submission based on just the Phase II and long-term studies.
FDA’s advisors recommended in a close 6-4 vote against approval using just the currently available data. The agency is expected to make a decision by June 29.
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