This is the reason behind many of the toughest Food and Drug Administration decisions, such as last year's controversial acceptanceof Aduhelm. Many experts, including outside advisors to the agency, believe that Alzheimer's drugs are unlikely to be of any benefit to patients.
The FDA may soon approve another drug to treat a deadly neurodegenerative disorder less than a year later. This approval could be based on partial data currently being discussed by experts. Next week, the FDA will meet to review evidence from a small and mid-stage study on Amylyx Pharmaceuticals' drug ALS (amytrophic lateral sclerosis).
Amylyx was told by regulators last year that it would need to conduct large-scale confirmatory studies before it could be approved. After months of lobbying by ALS patients, their representatives in Congress and other stakeholders, the agency decided to submit the drug based upon the smaller study.
Even doctors who studied the treatment were surprised by the sudden change. It appears to moderately slow down patients' decline.
Jeffrey Rothstein, Johns Hopkins University said that "the effect is there but it's no homerun." Does it work? It's not clear to me. This is why I would like to see another study.
Two large, late-stage studies are required by the FDA in order to be approved by the FDA. One study with promising early results is usually accepted for deadly diseases such as cancer.
The Amylyx decision is coming as government investigators examine Aduhelm’s approval. They also look at whether Aduhelm caved to pressure from pharmaceutical and Alzheimer's organizations.
Experts in FDA decision-making have identified a worrying pattern where the beleaguered agency is constantly pressured to accept weaker data, reducing its scientific credibility and opening up the doors to ineffective treatments.
Dr. Joseph Ross from Yale University stated that "this is what many people were worried about in terms of FDA approving Aduhelm." "They gave in to industry pressure and patient advocacy pressure rather than following the science.
A spokeswoman for FDA declined to discuss the review citing agency rules but stated that Amylyx's submission was not considered a decision on the merits of the application. FDA will publish its initial review of the drug before Wednesday's meeting.
The two drugs have important differences. The FDA approved the Alzheimer's drug on the basis of laboratory tests that suggested it could slow cognitive decline. However, company studies did not show any significant patient benefits. Amylyx's drug showed a measurable improvement in ALS patients, but no change in laboratory results.
Amylyx should be approved by the FDA because of its patient benefit.
Lou Gehrig's Disease, also known by ALS, is a disease that causes nerve cells to die. It can cause paralysis, speech, swallowing, and eventually, breathing problems. There is no cure, and most people will die within three to five year.
Amylyx's drug combines two older drug ingredients, a prescription medication to treat liver diseases and a dietary supplement that is associated with ancient Chinese medicine. Amylyx, a Cambridge, Mass. company that has patents the combination says the chemicals work together in order to protect cells from premature deaths. (Its cofounders declined interviews for this story.
Many ALS patients take both the pills. The FDA approval could force insurers to cover the treatment.
A 137-patient study found that people who took the drug performed 25% faster than those who did not. This was measured using a 48-point questionnaire, which tracks functions such as walking, handwriting, and swallowing. Although statistically significant, experts disagree on the meaning of this difference for patients.
Dr. Catherine Lomen Hoerth, University of California San Francisco, said that even one point could be significant.
Lomen-Hoerth, who was not involved in the research, said that "that's the difference between being capable of feeding yourself or not."
The New England Journal of Medicine editorial called the results "incremental and modest" and recommended larger, more detailed studies.
Although an FDA decision was years away, patient groups launched a campaign to get it done. The first was a petition asking for the agency's action. When FDA reversed its position on the medication in September, advocates took credit.
People with ALS should understand that any drug that prolongs or improves their lives is worth it.
Only two treatments for ALS have been approved by the FDA. The most effective treatment prolongs life by several more months. Experts believe Amylyx's drug will offer a similar benefit.
Larry Falivena was diagnosed with ALS in 2017 and said, "When you have to choose between nothing or something, you want the opportunity to try everything that can help." Next week, he'll be speaking at the FDA meeting.
The father of two, aged 53 years old, joined half a dozen other patients to call on FDA last May for Amylyx's approval.
The ALS Association organized the session, investing $2.2 million in Amylyx research. If the drug is approved, the proceeds could reach as high as $3.3 million for the group.
According to the association, such arrangements are common among non-profits that fund research. Proceeds go towards further studies. The group stated that payback clauses can be used to help bring "effective treatments to the market as quickly and efficiently as possible." It stated that it would not advocate for approval of a drug if it wasn't safe or effective.
During the listening session FDA's Dr. Patrizia Cazzoni reiterated its long-standing position that drugmakers could improve biological measures of the effectiveness of their medicines to speed up approvals.
Amylyx uses a 48-point scale, which is the standard for ALS research. However, experts are skeptical about its reliability. It's not unusual for patients to report improvements in function even when measures such as muscle strength decline.
Cavazzoni was summoned to a House of Representatives Subcommittee several weeks later. Patients' advocates requested that Cavazzoni be summoned before a a href="https://energycommerce.house.gov/committee-activity/hearings/hearing-on-the path-forward–advancing–treatments–and cures for">House of Representatives subcommittee. She was pressed by lawmakers about Amylyx's status.
I AM ALS was also founded by the co-founders. After Wallach's 2017 diagnosis of ALS, Brian Wallach and Sandra Abrevaya worked at the Obama White House. They founded their group. The non-profit has rapidly become a political force in Washington. It is pushing legislation that requires FDA to create a five year plan to accelerate drug development for ALS.
They called for lawmakers to intervene in the patient's behalf in their joint testimony, which Abrevaya read.
I AM ALS spokeswoman denied that the group "tells the FDA what to think."
Theresa Garner stated that "we are not trying pressure the FDA." "We are following the FDA's standard process and listening to people with this disease.
The FDA advisory panel, made up of outside experts, that met Wednesday was the same one who voted against Aduhelm. The FDA ignored its advice and approved Aduhelm. three of the members resigned.
Approval is expected to be granted because Amylyx is being overseen by the same FDA scientists as well as its leadership.
Rothstein, a Johns Hopkins researcher plans to prescribe the drug for his patients. He'd like to see more data.
He said, "I prefer that the FDA wait for at least two trials." "Patients will claim that the FDA is depriving them of drugs. But, the counter argument is that they may be being deprived of drugs that aren't as effective.