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Biogen/Ionis’ Tofersen Wins Accelerated Approval In ALS Subgroup – A Significant But Narrow Precedent

 
• By Sue Sutter

US FDA designates ongoing ATLAS trial in presymptomatic individuals with SOD1 mutation as confirmatory study. ‘Classic Biogen’ approval is first in ALS under the expedited pathway and could lead to more drugs reaching market on basis of a reduction in plasma neurofilament light chain concentration.

Footsteps on beach
Other ALS drug developers will look to follow Biogen/Ionis' lead in using reduction in neurofilament concentration as the basis for accelerated approval. • Source: Shutterstock

The US Food and Drug Administration opted for accelerated approval, rather than regular approval, for Biogen, Inc. and Ionis Pharmaceuticals, Inc.’s Qalsody (tofersen) in a genetically defined subset of amyotrophic lateral sclerosis patients, banking that clinical benefit will be confirmed in an ongoing trial of presymptomatic individuals.

The agency approved tofersen, an antisense oligonucleotide, on 25 April for treatment of ALS in adults who have a mutation...

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More from Approvals

J&J’s Inlexzo Is A Rare 505(b)(2) NDA To Qualify For US FDA’s Real-Time Oncology Review

 
• By Bridget Silverman

A Johnson & Johnson executive tells the Pink Sheet in an interview how the company developed its pretzel-shaped intravesical drug releasing system for gemcitabine, which used design and materials to improve efficacy and ease bladder cancer treatment.

Zimbabwe Publishes Public Assessment Reports In Line With International Practices

 
• By Eliza Slawther

The Medicines Control Authority of Zimbabwe has begun to publish public assessment reports for medicines it approves or rejects, in line with the World Health Organization’s highest maturity level benchmarking.

New EU Filings

 
• By Neena Brizmohun

Insulin efsitora alfa, Eli Lilly’s once-weekly, subcutaneous treatment for type 2 diabetes, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Japan Roundup: Approvals Recommended For Ibtrozi, Voranigo And Ycanth

 
• By Lisa Takagi

Along with multiple new therapeutics for NSCLC, Astellas's Izervay and several products licensed from US companies receive approval recommendations in Japan.

More from Product Reviews

First-In-Class Bronchiectasis Treatment Among 14 Drugs Awaiting EMA Verdict

 
• By Vibha Sharma

The European Medicines Agency is this week expected to recommend whether EU marketing approval should be granted to myriad new products, including therapies for bronchiectasis, RSV, menopause-related symptoms and type 2 diabetes.

More Vaccines At Risk At Upcoming ACIP Meeting As New Panel Members Join

 
• By Sarah Karlin-Smith

The ACIP put hepatitis B and MMRV vaccines on its agenda, despite having no new reason to relitigate the products, worrying public health experts that more anti-vaccine moves are coming.

US FDA Complete Response Letters Raise Confirmatory Evidence Shortfalls

 
• By Sue Sutter

The agency identified deficiencies in efficacy evidence intended to support results from a single adequate and well-controlled trial for orphan applications, including weaknesses in animal data and insufficient quantity and quality of biomarker data.