JOURNAL ARTICLE

First-of-its-kind drug for rare form of Lou Gehrig's disease gets FDA approval.

  • Published In: Fortune.com, 2023. P. N.PAG 1 of 3

  • Database: Business Source Ultimate 2 of 3

  • Authored By: Perrone, Matthew 3 of 3

Abstract

The article discusses the FDA's approval of tofersen, an injectable drug developed by Biogen for a rare genetic form of amyotrophic lateral sclerosis (ALS), which affects fewer than 500 people in the U.S. This marks the first drug approved for an inherited form of ALS, although the FDA has mandated further research to confirm its efficacy. The approval was granted through the FDA's accelerated pathway, which allows drugs to be launched based on early promising results, despite concerns about the robustness of the data. The ALS community has expressed hope that this approval could lead to more expedited treatments for the disease, which currently has no cure and significantly impacts patients' quality of life. [Extracted from the article]

Additional Information

  • Source:Fortune.com. 2023/04, pN.PAG
  • Document Type:Article
  • Subject Area:History
  • Publication Date:2023
  • Accession Number:163342156
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