FDA Grants Fast Track to Lundbeck's MSA Therapy Amlenetug

Lundbeck, a leading pharmaceutical company, has achieved a significant milestone with the US Food and Drug Administration (FDA) granting fast track designation for its investigational drug, amlenetug. This innovative treatment is aimed at addressing multiple system atrophy (MSA), a rare and debilitating neurological disorder. The fast track designation is a critical step that allows Lundbeck to benefit from rolling reviews and detailed guidance, thereby expediting the drug development process.

This development follows promising results from the Phase II AMULET trial, which highlighted the potential of amlenetug in treating MSA. Johan Luthman, Lundbeck's head of research and development and executive vice-president, expressed satisfaction with the FDA's decision, emphasizing the company's commitment to tackling significant unmet medical needs associated with MSA.

Amlenetug had previously garnered orphan drug designation in the United States and from the European Medicines Agency in April 2024 and May 2021, respectively. Additionally, the drug received Japan’s Ministry of Health, Labour and Welfare’s SAKIGAKE, or "pathfinder," designation in March 2023. These designations underscore the global recognition of the drug's potential impact on MSA treatment.

The drug is a human monoclonal antibody engineered to target and bind to extracellular α-synuclein. This action aims to inhibit its absorption and prevent aggregation, which is crucial in the pathology of MSA. Moreover, the drug's active Fc region might facilitate the immune-mediated clearance of α-synuclein through microglia-mediated uptake, enhancing its therapeutic efficacy.

Lundbeck is pursuing the development of amlenetug under a joint research and licensing agreement with Genmab, an established biotechnology company. This partnership underscores the collaborative effort to bring effective treatments to patients with neurodegenerative conditions. The drug is currently under evaluation in the Phase III MASCOT trial, which is designed to assess its safety and efficacy in individuals diagnosed with MSA. This trial is characterized as a randomized, interventional, double-blind, placebo-controlled, parallel-group study, with an optional open-label extension. It is being conducted across multiple regions, including Europe, North America, and Asia, to ensure comprehensive data collection and analysis.

In a strategic effort to boost its presence in the field of neuro-rare diseases, Lundbeck completed the acquisition of Longboard Pharmaceuticals in December 2024. This acquisition involved securing all outstanding shares of Longboard Pharmaceuticals, thereby consolidating Lundbeck's position and expanding its capabilities in developing treatments for rare neurological conditions.

The fast track designation for amlenetug marks a pivotal advancement in Lundbeck's journey to address the complex challenges of multiple system atrophy. With continued research and collaboration, the company is poised to make significant contributions to the field of neurodegenerative diseases, offering hope to patients and families affected by these challenging conditions.