Health Day News reports that despite months of intense lobbying by patient advocates, federal health officials on Monday posted a largely negative review of an experimental drug for the devastating illness known as amyotrophic lateral sclerosis (ALS).
In an analysis of Amylyx Pharmaceuticals’ drug, known for now only as AMX3005, regulators from the U.S. Food and Drug Administration said the results were “not persuasive.” They pointed to missing data, mistakes in enlisting patients and other problems. Meanwhile, the drug only modestly slowed the disease’s progression in a 137-patient, mid-stage study. An FDA advisory committee is set to meet Wednesday to consider Amylyx’s request to skip the typically required phase 3 clinical trial and approve its drug based on the earlier data.
Several dozen ALS patients and advocates plan to speak at the meeting, and the FDA will weigh the panel’s deliberations and vote before making a final ruling on the drug, which is expected by June. The push to approve the drug follows the agency’s controversial approval last year of the Alzheimer’s drug Aduhelm. Many of the FDA’s own advisors said at the time that the costly drug was unlikely to help patients.
But advocacy groups for patients with ALS are pushing hard for approval of AMX0035. They say the need is urgent. Most people with ALS, also known as Lou Gehrig’s disease, die of respiratory failure within three to five years, according to the U.S. National Institutes of Health. The disease destroys nerve cells needed to walk, talk, swallow and eventually breathe, steadily robbing patients of movement until they’re essentially “locked” inside their own bodies.
Amylyx’s request is supported by the ALS Association, one of the main advocacy and research groups devoted to finding a cure for this disease. “The community has been asking for a long time that the FDA approve the drug before that [phase 3] trial is complete,” said Neil Thakur, chief mission officer of the ALS Association. “And the reason why is because of a combination of strong clinical benefit and safety data that we’ve seen for this this drug.”
AMX0035 has been shown in early clinical trials to extend ALS patients’ lives by about six and a half months, Thakur said. The drug also slows disease progression in patients by about 25%, said Dr. James Berry, director of the Massachusetts General Hospital’s Neurological Clinical Research Institute in Boston and a principal investigator in the AMX0035 trials.
“ALS is a disease that doesn’t give us a lot of time to sort of stop and consider,” Berry said. “We really need to move when we see something that looks this promising without a safety concern and with a very well-run study that shows us both survival and function benefit.”
AMX0035 is a combination of two established drug ingredients — sodium phenylbutyrate and taurursodiol. Sodium phenylbutyrate is sold under the brand name Buphenyl to treat liver diseases, while taurursodiol is a dietary supplement used in ancient Chinese medicine. Some ALS patients already take both drugs. FDA approval would likely compel insurers to cover the treatment.
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