Much of the ALS drug news in recent months has focused on Amylyx Pharmaceuticals and its experimental program reviewed by an FDA advisory panel in March. But on Thursday, the FDA gave the green light to a new way to take an already approved ALS drug that has flown under the radar.
US regulators have approved an oral formulation of Radicava, first approved to treat ALS in 2017 as a series of intravenous 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.
The FDA granted approval to Mitsubishi Tanabe Pharma America, which applied for approval in January and received priority review.
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.
While riluzole can be taken by mouth, Radicava (edaravone) previously had to be given through multiple IV infusions. Patients had to travel to special ALS clinics or sites to receive the one-hour infusions for 14 consecutive days during the first 28-day cycle, and then 10 out of 14 days for each subsequent cycle.
Now patients can take the oral formulation themselves at home or via a feeding tube. The dosing schedule remains the same, the FDA said, but patients must also fast overnight before taking the oral version.
Radicava gained its first approval after it was able to show modest benefit in slowing functional decline, using a clinically validated but increasingly controversial measure. Mitsubishi Tanabe Pharma got the new OK thanks to a study showing comparable levels of the drug in patients’ blood to those seen from the IV formulation.
Thursday’s decision comes as patients have stepped up pressure on regulators in recent years, arguing the review process is too slow. The patient community scored a victory in 2019 when the FDA updated its guidelines for developing new ALS drugs, including a provision advising against withholding approved treatments in placebo arms.
More recently, patients have rallied around the drug Amylyx in hopes of swaying FDA experts. At the Amylyx adcomm earlier this year, approximately 30 patients spoke during the nearly two-hour public comment period, highlighting the need for new drug approvals for ALS.
It remains to be seen whether regulators will give the green light to the Amylyx program, dubbed AMX0035. Although the drug reached statistical significance in a phase II study measuring function and appeared to show some survival benefit in long-term follow-up, open questions remained about the quality of this latest data set.
However, the FDA has been more open to reviewing the drug Amylyx. After initially asking the biotech to conduct a large-scale phase III trial before submitting its NDA, regulators changed their minds and allowed the submission based only on phase II and long-term studies.
FDA advisers recommended in a close 6-4 vote against approval using only currently available data. The agency is expected to issue a decision by June 29.