Amlenetug Receives FDA Fast Track Designation for Multiple System Atrophy
Lundbeck’s investigational drug, amlenetug, has been granted Fast Track Designation by the US Food and Drug Administration (FDA) as a potential treatment for multiple system atrophy (MSA).
This designation underscores the urgency of developing therapies for MSA, a rare and aggressive neurodegenerative disorder with limited treatment options. The FDA’s decision follows encouraging findings from the AMULET phase 2 trial, presented in March 2024, which demonstrated promising results in slowing disease progression.
Amlenetug is a human monoclonal antibody designed to target all major forms of extracellular α-synuclein, a protein implicated in MSA pathogenesis.
By preventing the uptake and aggregation of α-synuclein, amlenetug aims to address one of the critical factors contributing to disease progression. To further evaluate its efficacy and safety, Lundbeck has initiated the MASCOT phase 3 trial, an extensive study designed to build upon the findings of AMULET.
Lundbeck’s Executive Vice President and Head of Research & Development expressed confidence in the potential of amlenetug and emphasised that the Fast Track Designation reflects the company’s commitment to addressing significant unmet needs in MSA treatment.
Furthermore, the FDA’s Fast Track process is intended to facilitate the development and expedite the review of promising therapies for serious conditions lacking effective treatments.
Amlenetug has also received Orphan Drug Designation (ODD) from multiple regulatory agencies, including the US FDA in April 2024, the European Medicines Agency (EMA) in May 2021, and Japan’s Ministry of Health, Labor and Welfare through its SAKIGAKE designation in March 2023.
The FDA grants ODD to therapies developed for rare diseases affecting fewer than 200,000 individuals in the United States, further underscoring amlenetug’s potential impact in the MSA treatment landscape.
The ongoing MASCOT phase 3 trial is a large-scale interventional study that will randomise participants to receive either high or low doses of amlenetug or a placebo for 72 weeks. Following this, patients will enter an open-label extension period to assess long-term effects.
The trial’s primary objective is to determine the drug’s efficacy in slowing disease progression, while secondary outcomes will evaluate patient functioning, disease severity, and overall tolerability. Amlenetug is administered intravenously every four weeks.
The AMULET phase 2 trial, which set the foundation for the phase 3 study, involved 61 patients who received either amlenetug or a placebo over a period of 48 to 72 weeks, followed by a 96-week open-label extension.
The primary goal was to demonstrate a reduction in clinical progression, with secondary measures focusing on functional outcomes, disease burden, and overall patient response.
With the initiation of the MASCOT phase 3 trial and the FDA’s Fast Track Designation, amlenetug is positioned as a promising candidate in the fight against MSA. Lundbeck’s continued research and regulatory progress signal a potential breakthrough for patients affected by this devastating condition.
If successful, amlenetug could provide new hope and a much-needed therapeutic option for those living with MSA.
News Credits: Amlenetug receives FDA Fast Track Designation
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