Good Health Medicine

Main Menu

  • Home
  • Health medicine
  • Dietary supplements
  • Dietary nutrition
  • Alternative medication
  • Medicine price

Good Health Medicine

Header Banner

Good Health Medicine

  • Home
  • Health medicine
  • Dietary supplements
  • Dietary nutrition
  • Alternative medication
  • Medicine price
Health medicine
Home›Health medicine›FDA skeptical of benefits of experimental ALS drug | Health, Medicine and Fitness

FDA skeptical of benefits of experimental ALS drug | Health, Medicine and Fitness

By Rebecca C. Myers
March 28, 2022
0
0

By MATTHEW PERRONE – AP Health Writer

WASHINGTON (AP) — Federal health regulators on Monday released a negative review of a closely watched experimental drug for the wasting disease known as Lou Gehrig’s disease, after months of lobbying by patient advocates asking for its approval.

Amylyx Pharmaceuticals’ drug has become a rallying cause for patients with the deadly neurodegenerative disease ALS, their families and members of Congress who have joined in pushing the FDA to greenlight the drug.

But regulators said in a review that the company’s small study was “unconvincing,” due to missing data, errors in patient recruitment and other issues. On Wednesday, a panel of FDA advisers will hold a nonbinding vote on whether the drug merits approval.

The meeting is being closely watched as an indicator of the FDA’s approach to investigational drugs with imperfect data and its ability to withstand outside pressure.

The FDA’s negative stance on the drug creates a tense scenario at Wednesday’s town hall meeting, where several dozen ALS patients and advocates are scheduled to speak. The agency will consider feedback from its advisory committee before making a final decision on the drug, expected by June.

ALS, or amyotrophic lateral sclerosis, destroys the nerve cells needed to walk, talk, swallow and, eventually, breathe. There is no cure and most people die within three to five years.

Amylyx’s drug is a combination of two older drugs: a prescription drug for liver disorders and a dietary supplement associated with traditional Chinese medicine. Amylyx has patented the combination and claims the chemicals help protect cells from early death.

But FDA reviewers found the drug had “only a modest effect” in slowing disease progression in a mid-term study of 137 patients, who the FDA said was plagued to problems of implementation and analysis. Typically, FDA approval requires two large studies or one study with a “very persuasive” effect on survival.

According to the FDA document, regulators had “strongly suggested” that Amylyx complete a large, late-stage trial to establish the drug’s effectiveness before rolling it out. This study should be completed in 2024.

After further discussion, Amylyx decided to submit its pill for approval based on survival data collected after its initial study. The company said this showed patients taking the drug lived about six months longer than those taking a dummy pill.

But the FDA says the data is unreliable because of problems tracking study participants for several years.

Wednesday’s meeting marks the first time the FDA has convened this advisory group since ignoring its advice last June and approving Biogen’s advice. Alzheimer’s disease drug, Aduhelm. Three panel members resigned over the controversial decision, and congressional investigators have launched an investigation into the FDA’s handling of the drug’s review. As in the current case, the FDA faced intense pressure from patient groups to grant approval.

Currently, there are only two FDA-approved drugs for ALS, the most effective of which prolongs life by several months.

The ALS Association, which helped fund the Amylyx research, said the FDA review does not take into account “the rapidity and severity of ALS and the few treatment options available.”

“It’s a uniformly fatal disease, so any drug that has that benefit – and even if that benefit is modest – is fantastic for patients, who have only one way to go with this disease: death. “said Dr. Jeffrey Rothstein of Johns Hopkins University, who helped enroll patients in the Amylyx study.

Shares of the Cambridge, Mass.-based drugmaker fell more than 50% in morning trading after the FDA review.

The Associated Press Health and Science Department is supported by the Howard Hughes Medical Institute Department of Science Education. The AP is solely responsible for all content.

Copyright 2022 The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed without permission.

Sign up here to get the latest health and fitness updates delivered to your inbox every week!

Related posts:

  1. Living with a Short Sleep Gene: “It’s a Gift” | Health, medicine and fitness
  2. COVID counts remain low in Nebraska; vaccination figures fall | Health, medicine and fitness
  3. The Best Over-The-Counter Allergy Relief Options Health, medicine and fitness
  4. FACTS FOR FAMILIES: Where to get your vitamin D | Health, medicine and fitness
  • Alternative medication
  • Dietary nutrition
  • Dietary supplements
  • Health medicine
  • Medicine price
  • Survey compares how consumers and nutrition experts rate healthy foods – AgriNews
  • Bone & Joint Health Supplements Market to Witness a CAGR of 8% (2022-2028) Driven by Growing Prevalence of Orthopedic Disorders and Micronutrient Deficiencies | UnivDatos Market Overview
  • Police arrest scammer entangling hundreds of IPB students in online loans
  • Know which one is better for nutrition, skin and health
  • Your answers on cancer: What to know about stomach cancer? | Health, Medicine and Fitness
  • November 2022
  • October 2022
  • September 2022
  • August 2022
  • July 2022
  • June 2022
  • May 2022
  • April 2022
  • March 2022
  • February 2022
  • January 2022
  • December 2021
  • November 2021
  • October 2021
  • September 2021
  • August 2021
  • July 2021
  • June 2021
  • May 2021
  • April 2021
  • March 2021
  • February 2021
  • January 2021
  • December 2020
  • November 2020
  • October 2020
  • August 2020
  • May 2020
  • March 2020
  • December 2019
  • November 2019
  • October 2019
  • September 2019
  • August 2019
  • June 2019
  • May 2019
  • April 2019
  • March 2019
  • February 2019
  • January 2019
  • December 2018
  • November 2018
  • October 2018
  • September 2018
  • August 2018
  • July 2018
  • June 2018
  • March 2018
  • February 2018
  • January 2018
  • November 2017
  • October 2017
  • September 2017
  • August 2017
  • August 2016
  • June 2016
  • May 2016
  • February 2016
  • October 2015
  • August 2015
  • April 2015
  • March 2014
  • December 2013
  • September 2012
  • October 2011
  • September 2011
  • July 2011
  • June 2011
  • April 2011
  • March 2011
  • March 2010
  • September 2007
  • Privacy Policy
  • Terms and Conditions