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☆ A.L.S. Drug Relyvrio Will Be Taken Off the Market, Its Maker Says
▼ + stars: | 2024-04-04 | by ( Pam Belluck | ) www.nytimes.com time to read: +1 min

The maker of the newest treatment approved for amyotrophic lateral sclerosis said Thursday that it would withdraw the drug from the market because a large clinical trial did not produce evidence that the treatment worked. The company, Amylyx Pharmaceuticals, said in a statement that it had started the process of withdrawing the drug in the United States, where it is called Relyvrio, and in Canada, where it is called Albrioza. As of Thursday, no new patients will be able to start the drug, while current patients who wish to continue taking the medication can be transitioned to a free drug program, the company said. It decided to greenlight the medication anyway, instead of waiting two years for results of a large clinical trial, citing data showing the treatment to be safe and the desperation of A.L.S. The disease robs patients of their ability to control muscles, speak and breathe without assistance and often causes death in two to five years.

Organizations: Amylyx Pharmaceuticals, and Drug Administration Locations: United States, Canada

☆ A.L.S. Drug Relyvrio Fails Clinical Trial and May Be Withdrawn From the Market
▼ + stars: | 2024-03-08 | by ( Pam Belluck | ) www.nytimes.com time to read: +1 min

One of the few treatments the Food and Drug Administration has approved for amyotrophic lateral sclerosis has failed a large clinical trial, and its manufacturer said Friday that it was considering whether to withdraw it from the market. The medication, called Relyvrio, was approved less than two years ago, despite questions about its effectiveness in treating the severe neurological disorder. At the time, the F.D.A.’s reviewers had concluded there was not yet sufficient evidence that the medication could help patients live longer or slow the rate at which they lose functions like muscle control, speaking or breathing without assistance. But the agency decided to greenlight the medication instead of waiting two years for results of a large clinical trial, citing data showing the treatment to be safe and the desperation of patients with a disease that often causes death within two to five years. Now, results of the 48-week trial of 664 patients are in, and they showed that the treatment did not work better than a placebo.

Persons: ’ Organizations: and Drug Administration Locations: United States

Amylyx said Friday it would discuss its plans for Relyvrio with patients and the FDA, which “may include voluntarily withdrawing” the drug. The latest company study showed that the drug did not slow the disease compared with a dummy treatment. Amylyx’s medication is part of a string of drugs for deadly, degenerative diseases that have won FDA approval in recent years despite questionable proof that they work. That commitment seemed to reassure FDA's advisers who voted in favor of the drug's approval, despite the questionable data. In the months before the decision the FDA faced intense pressure from ALS patients, advocates and members of Congress.

Persons: Lou Gehrig’s, Amylyx, ”, “, Relyvrio Organizations: WASHINGTON, Drug Administration, Amylyx Pharmaceuticals, Relyvrio, FDA, Cambridge, Amylyx Pharmaceuticals Inc, Associated Press Health, Science Department, Howard Hughes Medical Institute’s Science, Educational Media Group, AP Locations: U.S

☆ FDA staff reviewers raise concerns over BrainStorm Cell's ALS therapy
▼ + stars: | 2023-09-25 | by ( ) www.reuters.com time to read: +2 min

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. Brainstrom's therapy, NurOwn, is being reviewed for the treatment of ALS, also known as Lou Gehrig's Disease, which affects nerve cells in the brain and spinal cord. The FDA had in November last year refused to accept the application for the therapy after BrainStorm failed to meet the main goal in a late-stage study. Over the last one year, the FDA has given accelerated approvals to Amylyx Pharmaceuticals' (AMLX.O) drug Relyvrio and Biogen's (BIIB.O) Qalsody for treatment of ALS. A panel of external advisors to the FDA is set to vote on the effectiveness of the therapy in ALS patients on Wednesday.

Persons: Andrew Kelly, Lou Gehrig's Disease, Amylyx, Mariam Sunny, Leroy Leo, Krishna Chandra Organizations: Food and Drug Administration, FDA, REUTERS, U.S . Food, Drug, Cell Therapeutics, Amylyx Pharmaceuticals, ALS Association, Thomson Locations: White Oak , Maryland, U.S, Bengaluru

Those advocates still face one giant hurdle: FDA regulators say the treatment hasn't been shown to work. But with the backing of thousands of ALS patients, Brainstorm took the rare step of “filing over protest,” essentially forcing the agency to render a decision. Still, ALS patients see reasons for optimism. At Wednesday’s meeting, people with such reservations are certain to be outnumbered by appeals from ALS patients and their families. That compromise would be similar to FDA's approach to Relyvrio last year, another ALS drug with questionable data.

Persons: Lou Gehrig’s, drugmaker, they’re, ”, Marc Scheineson, Brian Wallach, Wallach, “, Brainstorm’s, NurOwn, Patricia Manhardt, Anthony Windebank, reanalyzing, weren’t, Terry Heiman, Patterson, it’s, Holly Fernandez Lynch, Matthew Perrone Organizations: WASHINGTON, Drug Administration, FDA, drugmakers, Congress, Obama White House, ALS Association, Mayo Clinic, Physicians, Temple University, University of Pennsylvania, Twitter, Associated Press Health, Science Department, Howard Hughes Medical Institute’s Science, Educational Media Group, AP

☆ Goldman upgrades this biotech, sees the shares doubling
▼ + stars: | 2023-07-24 | by ( Alex Harring | ) www.cnbc.com time to read: +2 min

It's a good time to buy into beat-down Amylyx Pharmaceuticals as investors could be underestimating one of its trials, according to Goldman Sachs. Analyst Corinne Jenkins upgraded the pharmaceutical stock to buy from neutral and increased her price target by $4 to $49. Her new price target implies shares can surge 117% from Friday's close. But investors have been concerned about the stock because of low expectations going into the phase 3 Phoenix study for Relyvrio. But Jenkins also said the approval would likely be a positive catalyst for the beat-down stock.

Persons: Goldman Sachs, Corinne Jenkins, Jenkins, Lou Gehrig's, — CNBC's Michael Bloom Organizations: Pharmaceuticals, PHOENIX Locations: Friday's, U.S

☆ Here are Monday's biggest analyst calls: Apple, Tesla, Meta, Nvidia, Shopify, Netflix, Disney & more
▼ + stars: | 2023-07-24 | by ( Michael Bloom | ) www.cnbc.com time to read: +9 min

Goldman Sachs initiates Amylyx Pharmaceuticals as buy Goldman said it sees an attractive entry point for shares of the biotech company. " Goldman Sachs reiterates Nvidia as buy Goldman said it's standing by its buy rating on Nvidia and that it likes stocks with "room for meaningful positive EPS revisions." UBS downgrades Tesla to neutral from buy UBS said in its downgrade of Tesla that strong growth is already priced in. Goldman Sachs initiates Savers Value Village as buy Goldman said the thrift store operator is a share gainer. Goldman Sachs reiterates ServiceNow as buy Goldman said it sees more upside in shares of ServiceNow as the stock remains a key beneficiary.

Persons: Piper downgrades Estee Lauder, Piper, Baird, Goldman Sachs, Goldman, Wells, Evercore, it's, Stifel, Lam, Tesla, Moffett, Rosenblatt, Raymond James, Becton Dickinson, SLB, Horton, BTIG, Needham, ServiceNow Organizations: Express, Netflix, Amylyx Pharmaceuticals, Bank of America, NTR, RBC, Research, Nvidia, MU, UBS, Semiconductor, Deutsche Bank, Apple, Deutsche, Mac, Med Tech, Schlumberger, Disney, DIS, American Express Locations: China, underperform, ServiceNow

☆ Citi names its top biotech and pharma stock picks, giving one nearly 90% upside
▼ + stars: | 2023-05-17 | by ( Weizhen Tan | ) www.cnbc.com time to read: +2 min

The healthcare sector outperformed in Europe, India and China last month, according to Citi, which named its top picks worldwide. It gave Amylyx a price target of $53, giving it upside of around 88% from current levels. It gave Beam a price target of $60, implying upside of around 67%. Other European picks from Citi include Swiss healthcare company Roche and British pharma and biotech firm AstraZeneca . Asia stocks In Asia, Citi named Japanese pharmaceutical company Daiichi Sankyo , South Korean biotech firm Samsung Biologics and China's Wuxi Biologics as top picks.

☆ FDA neurosciences chief Billy Dunn to leave immediately, analysts raise concern
▼ + stars: | 2023-02-28 | by ( ) www.reuters.com time to read: +2 min

Feb 28 (Reuters) - The U.S. Food and Drug Administration neurosciences head Billy Dunn will retire from his role effective immediately, the health regulator told Reuters on Tuesday. Wall Street analysts said Dunn's departure could impact the regulator's stance on neurological drug decisions in the near term, sending down shares of Reata Pharmaceuticals Inc (RETA.O) 30% on Monday when media reports on the exit surfaced. Texas-based Reata is expecting FDA's decision by Tuesday on its drug to treat a rare neuromuscular disorder called Friedreich's ataxia. The departure of Dunn, 53, was announced in an internal message to the agency's employees on Monday. Newly appointed deputy director Teresa Buracchio will also serve in Dunn's role on an interim basis at the Office of Neuroscience, the agency told Reuters.

☆ Why a $158,000 Drug With Unclear Benefits Hurts Whole Health System
▼ + stars: | 2022-10-10 | by ( David Wainer | ) www.wsj.com time to read: 1 min

Like many patients suffering from amyotrophic lateral sclerosis, or ALS—also known as Lou Gehrig’s disease—Layne Oliff didn’t have any time to waste. Even before the drug Relyvrio was approved late last month by the Food and Drug Administration, he has had his own do-it-yourself method: he gets sodium phenylbutyrate in liquid form from a New Jersey pharmacy and taurursodiol online from Amazon. That costs him over $7,000 a year, but he says it has been well worth it because he feels the combination has helped stabilize a disease that often causes death within a few years.

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