--- title: FDA Approves Drug to Treat Rare Form of ALS description: Biogen's drug, Qalsody, was cleared through an accelerated approval pathway, meaning more research is needed for it to stay on the market. canonical: https://time.com/6274513/fda-approves-als-drug/ author: TIME article:opinion: false article:content_tier: free article:published_time: 2023-04-25T20:47:19.000Z article:modified_time: 2026-02-24T15:47:01.480Z article:section: Health og:title: FDA Approves Drug to Treat Rare Form of ALS og:description: Biogen's drug, Qalsody, was cleared through an accelerated approval pathway, meaning more research is needed for it to stay on the market og:url: https://time.com/6274513/fda-approves-als-drug/ og:site_name: TIME og:image: https://static.time.com/v3/assets/bltea6093859af6183b/blt3cb7ef26c5b65c16/698a3c4822fb85448be2e9c9/fda-approves-als-drug.jpg?branch=production&width=3840&quality=75&auto=webp&crop=16:9 og:image:width: 1200 og:image:height: 675 og:image:alt: Biogen og:type: article twitter:card: summary_large_image twitter:title: FDA Approves Drug to Treat Rare Form of ALS twitter:description: Biogen's drug, Qalsody, was cleared through an accelerated approval pathway, meaning more research is needed for it to stay on the market twitter:image: https://static.time.com/v3/assets/bltea6093859af6183b/blt3cb7ef26c5b65c16/698a3c4822fb85448be2e9c9/fda-approves-als-drug.jpg?branch=production&width=3840&quality=75&auto=webp&crop=16:9 ---  # FDA Approves Drug to Treat Rare Form of ALS by [Fiona Rutherford and Robert Langreth/Bloomberg](https://time.com/author/bloomberg/) ## Fiona Rutherford and Robert Langreth/Bloomberg Apr 25, 2023 8:47 PM UTC  Signage outside the Biogen Inc. office in the Kendall Square neighborhood of Cambridge, Massachusetts, US, on Tuesday, Sept. 6, 2022\. The regional growth of the biomanufacturing industry in Massachusetts led to significant year-over-year employment growth compared with competitor states, according to the 2022 Massachusetts Biotechnology Council Industry Snapshot report. Photographer: Adam Glanzman/Bloomberg via Getty Images Signage outside the Biogen Inc. office in the Kendall Square neighborhood of Cambridge, Massachusetts, US, on Tuesday, Sept. 6, 2022\. The regional growth of the biomanufacturing industry in Massachusetts led to significant year-over-year employment growth compared with competitor states, according to the 2022 Massachusetts Biotechnology Council Industry Snapshot report. Photographer: Adam Glanzman/Bloomberg via Getty ImagesAdam Glanzman—Bloomberg/Getty Images by [Fiona Rutherford and Robert Langreth/Bloomberg](https://time.com/author/bloomberg/) ## Fiona Rutherford and Robert Langreth/Bloomberg Apr 25, 2023 8:47 PM UTC Biogen Inc.’s drug for a rare form of amyotrophic lateral sclerosis was approved by U.S. regulators, marking the first clearance of a treatment targeting a specific genetic cause of the incurable paralysis disease. The Food and Drug Administration granted clearance for the drug, called Qalsody, through an accelerated approval pathway, meaning Biogen must conduct more research to establish its benefits for it to remain on the market. The approval was disclosed on the agency’s website Tuesday. ALS, sometimes called Lou Gehrig’s disease, destroys nerve cells that control voluntary muscle movement, including breathing. Around 31,000 people in the U.S. are living with the disease, according to Centers for Disease Control and Prevention [estimates](https://www.cdc.gov/als/WhatisALS.html). Roughly 5,000 new patients are diagnosed with ALS each year. The FDA estimated there are fewer than 500 patients in the U.S. with the form of ALS that the Biogen drug treats. Qalsody’s clearance has been a challenging journey for Biogen and partner Ionis Pharmaceuticals Inc. After a late-stage trial failed to show that the drug slowed the disease, the companies didn’t conduct a second, larger trial to confirm whether the drug worked. A second trial would “not appear to be feasible” given the rarity of the disease, FDA staff said in a report in March. Instead, Biogen applied for clearance through the accelerated approval pathway on the basis that the drug reduces levels of a blood protein associated with brain injury. In an advisory committee meeting in March, Qalsody, formerly known as tofersen, got partial support from an independent panel of advisers. They said the company hadn’t proven the drug worked but they agreed that reducing proteins was a promising sign that the drug would help patients. “Today also marks a pivotal moment in ALS research,” said Christopher Viehbacher, Biogen’s chief executive officer, in an emailed statement after the approval. “We believe this important scientific advancement will further accelerate innovative drug development for ALS.” Shares of Biogen fell 3.5% at 2:03 p.m. in New York, while Ionis climbed as much as 0.5%. ```json [{"@context":"https://schema.org","@type":"NewsArticle","@id":"https://time.com/6274513/fda-approves-als-drug/","mainEntityOfPage":{"@type":"WebPage","@id":"https://time.com/6274513/fda-approves-als-drug/"},"headline":"FDA Approves Drug to Treat Rare Form of ALS","datePublished":"2023-04-25T20:47:19.000Z","dateModified":"2026-02-24T15:47:01.480Z","description":"Biogen's drug, Qalsody, was cleared through an accelerated approval pathway, meaning more research is needed for it to stay on the market.","url":"https://time.com/6274513/fda-approves-als-drug/","keywords":["Drugs","wire","healthscienceclimate","bloomberg wire"],"thumbnailUrl":"https://static.time.com/v3/assets/bltea6093859af6183b/blt3cb7ef26c5b65c16/698a3c4822fb85448be2e9c9/fda-approves-als-drug.jpg?branch=production&width=1200&quality=75&auto=webp&crop=1200:675&height=675","author":[{"@type":"Person","name":"Fiona Rutherford and Robert Langreth/Bloomberg"}],"articleSection":"Health","image":[{"@type":"ImageObject","url":"https://static.time.com/v3/assets/bltea6093859af6183b/blt3cb7ef26c5b65c16/698a3c4822fb85448be2e9c9/fda-approves-als-drug.jpg?branch=production&width=1200&quality=75&auto=webp&crop=1200:675&height=675","width":1200,"height":675,"headline":"Biogen","caption":"Biogen","creditText":"Adam Glanzman—Bloomberg/Getty Images","representativeOfPage":true}],"publisher":{"@type":"Organization","name":"Time","url":"https://time.com/","logo":{"@type":"ImageObject","url":"https://time.com/images/logo.png","width":528,"height":156},"foundingDate":"March 3, 1923","sameAs":["https://www.facebook.com/time","https://www.instagram.com/time/?hl=en","https://twitter.com/time","https://www.pinterest.com/timemagazine"]}},{"@context":"https://schema.org","@type":"BreadcrumbList","itemListElement":[{"@type":"ListItem","position":1,"item":{"@id":"/section/health/","name":"Health"}},{"@type":"ListItem","position":2,"item":{"@id":"/tag/drugs/","name":"Drugs"}},{"@type":"ListItem","position":3,"item":{"@id":"https://time.com/6274513/fda-approves-als-drug/","name":"FDA Approves Drug to Treat Rare Form of ALS"}}]}] ```