ALS Medication Receives FDA Approval Despite Dubious Data

A hotly contested treatment for Lou Gehrig’s illness received U.S. approval on Thursday, a long-awaited win for sufferers likely to raise new concerns about the validity of the science supporting government assessments of experimental drugs.

FDA Approved

Based on findings from tiny, mid-stage research in which patients with the crippling condition appeared to proceed more slowly and lived several months longer, the Food and Medication Administration approved the Amylyx Pharmaceuticals drug. The FDA typically demands two extensive studies or one trial with “extremely compelling” survival findings for approval.

The FDA’s neurology drug director Dr. Billy Dunn stated that “this clearance gives another crucial therapeutic option for ALS, a life-threatening disease that currently has no cure.”

Relyvrio is the third ALS treatment to receive U.S. approval. ALS attacks the nerve cells necessary for essential bodily activities like walking, talking, and swallowing. The condition affects about 20,000 persons in the United States.

The FDA’s examination has emerged as a hot topic in more extensive discussions about the regulatory body, including how accommodating it should be when evaluating medications for fatal conditions and how much weight it ought to, according to the patient and other outside voices.

Dr. Catherine Lomen-Hoerth, an expert on ALS at the University of California San Francisco, said, “I believe it highlights the FDA’s propensity to be facile, and I think it demonstrates a lot of tenacity on the part of ALS patients and activists.” The business made every effort to provide this potentially effective medication to patients.

The medication from Amylyx is the most recent in a line of neurological medications that have received FDA approval despite conflicting effectiveness data. Two government investigations investigating the agency’s last year’s approval of the Alzheimer’s medicine Aduhelm, which has not yet been demonstrated to delay the illness, are still ongoing.

Given ALS’s severity and life-threatening nature and the significant unmet demand, the FDA determined that “regulatory flexibility” was justified for authorizing Relyvrio in an online document outlining its decision.

The FDA’s internal scientists gave the company’s results two unfavorable reviews, describing them as “borderline” and “not compelling,” until the most recent approval came about. In March, a group of independent advisors decided against the medicine, supporting that unfavorable opinion.

But ALS patients, supporters, and Congressmen have put significant pressure on the FDA. More than 1,300 written comments in support of the treatment were sent to the agency in recent weeks by members of the ALS community.

When the FDA reassembled the same expert group earlier this month to discuss Amylyx’s medication, that outpouring influenced them. They favored the drug 7-2 the second time. Although the decision was not legally binding, it appeared to pave the way for FDA clearance.

The FDA’s Dr. Dunn requested — and Amylyx confirmed — that the company will voluntarily remove its medicine from the market if sizable, ongoing research doesn’t demonstrate its value. Several panelists stated this conversation gave them more peace of mind.

600 patient report

Results from a 600-patient study are anticipated in 2024. Experts have noted the numerous issues that such a loose commitment could cause. If the FDA and the company disagree about whether the final results support the drug, or if another company later buys the drug, they might not feel obligated to uphold Amylyx’s promise.

A prescription pill for liver conditions and a nutritional supplement linked to traditional Chinese medicine make up the powder-based remedy, which combines two older substances. Based in Cambridge, Massachusetts, The mixture has been patented by Amylyx, which claims that the compounds act together to protect cells from dying too soon.

According to doctors, some ALS patients already take both medications separately and occasionally pay $5,000 a month for the prescription portion. Insurance companies are anticipated to cover Amylyx’s prescription following FDA clearance.

Price to be Announced

Amylyx claimed Thursday that it would announce the pricing on a call with investors on Friday morning but did not do so right away. The business has suggested a price of $165,000 for the medicine in Canada, where it was approved in June.

The drug’s worth was estimated by one external organization that examines the cost-effectiveness of novel treatments to be between $9,100 to $30,700 annually. The Institute for Clinical and Economic Review also pointed out that many patients will likely combine Amylyx’s medication with an earlier therapy, which has an annual cost of more than $170,000.

Sunny Brous, who received her ALS diagnosis in 2015, intends to supplement the two existing ALS drugs she is now taking with Relyvrio.

The 35-year-old Brous, who lives close to Fort Worth, Texas, said, “I’ve made it this far without this drug, and I’m hopeful using it now will extend my life further.”

Based on patient functionality questionnaires from a 6-month study involving 137 patients, Amylyx data suggested some effectiveness in delaying the disease. According to the FDA, patients who continued using Relyvrio after the study looked to live longer than those who first received a placebo.

Response

In a statement sent via email, Larry Falivena, an ALS Association board member diagnosed with the illness in 2017, said, “The ALS community has proved that our advocacy can affect decisions being made concerning our health.” The organization contributed $2.2 million to the early study of Amylyx and hopes to gain $3.3 million from medicine sales.

 

Gloria Flynt

I am a Research Content Specialist in Update.co.ke. I have been working with update.co.ke for over 6 months. Update.co.ke is a digital platform that provides news and analysis on business, economy, technology and entrepreneurship in worldwide. I love reading and writing about anything that has to do with science, technology, and developments in the digital world.

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