The closely watched experimental drug for Lou Gehrig’s disease received an unusual second glance from US regulators on Wednesday after heavy pressure to approve treatment for people with the terminal disease.
Patients and their families teamed up with Amylyx Pharma for the drug, starting an aggressive lobbying campaign and recruiting members of Congress to urge the Food and Drug Administration to grant approval.
The FDA has approved only two treatments for the disease, amyotrophic lateral sclerosis, which destroys nerve cells needed for basic functions such as walking, speaking, and swallowing. The more effective of the two drugs prolongs life by several months.
In a rare move, the FDA called a second meeting in March of neuroscientists who narrowly voted against the company’s drug. The panel was reviewing Amylyx’s new statistical analyzes and planned to re-vote or recommend approval. The FDA is not obligated to follow group guidelines.
An in-house review by FDA researchers prior to the meeting struck a negative note, stating that the company’s updated analysis was not “convincing” and “did not contain any new data.”
On the other hand, the FDA’s instructions to the panel emphasized the need for regulatory flexibility when considering drugs for terminal diseases.
The final decision of the FDA is expected later this month.
dr. Billy Dunn, the FDA’s director of neurological review, opened the meeting by noting “concerns and limitations” in the Amylyx data while stressing the need for new options for patients.
“We are very sensitive to the urgent need to develop new treatments for ALS,” said Dunn. “We have not taken any final decisions on the admissibility of this application.” Dunn also noted that a larger study by Amylyx could provide “more conclusive results” on the drug’s effectiveness by 2024.
ALS drug review is being closely watched as an indicator of the FDA’s flexibility in reviewing experimental drugs for terminally ill and their ability to withstand outside pressure.
“We’re here because there’s a lot of pressure,” said Diana Zuckerman of the non-profit National Center for Health Research, which analyzes data and conducts medical research.
“The FDA goes one step further by saying you may have another meeting, but the company responded without giving them new data.” Amylyx did one small, middle study of its drug that showed some benefits in slowing the disease, but was plagued by missing data and other problems, according to FDA reviewers.
The Cambridge, Massachusetts-based company says data collected after the study showed the drug extended life. Patients who continued taking the drug lived about 10 months longer than patients who never took the drug, according to a new study by the company.
But FDA scientists said in their review that the new approach “suffers from the same interpretation challenges” as the original study.
The FDA will hear again on Wednesday from patients and advocacy groups such as I AM ALS, which has been lobbying for more than two years to release the drug. “We have a drug that is driven by the entire ALS community. Patients, clinicians, and researchers support this because of what we saw in the study data, ”said I AM ALS co-founder Brian Wallach, speaking through an interpreter. “This is not the case with every drug.” Amylyx comes as a powder that combines two older medicines: one prescription medicine for liver disorders and a dietary supplement used in traditional Chinese medicine. Wallach and some other ALS patients are already taking the dietary supplement.
Hanging over the review was the controversial FDA approval of the Alzheimer’s drug Aduhelm last year, which was reviewed by researchers from the same agency and external advisers.
In this case, the FDA ignored the overwhelmingly negative vote of its external advisers, three of whom dropped the decision. FDA approval – which followed irregular meetings with drug manufacturer Biogen – is being investigated by congressional committees and federal inspectors.
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