FDA Grants Fast Track Status to Lundbeck's Amlenetug for Multiple System Atrophy

Lundbeck, a renowned biopharmaceutical company, has announced a significant achievement in the development of its investigational drug, amlenetug. This drug has recently been granted Fast Track designation by the United States Food and Drug Administration (FDA) as a potential treatment for Multiple System Atrophy (MSA), a rare and debilitating neurological disorder. This designation is based on promising results from the phase II AMULET trial, which were presented in March 2024 at the International Conference on Alzheimer's and Parkinson's Diseases and related neurological disorders in Lisbon, Portugal.

The Fast Track designation from the FDA is a crucial step in accelerating the development and review process of amlenetug, allowing Lundbeck to benefit from expedited reviews and guidance on drug development. Johan Luthman, Executive Vice President and Head of Research & Development at Lundbeck, expressed his satisfaction with this progress, stating that it is a vital step towards addressing the unmet needs of patients suffering from MSA.

In addition to the Fast Track status, amlenetug has been awarded Orphan Drug Designation by the FDA and the European Medicines Agency (EMA) for the treatment of MSA, as well as SAKIGAKE designation from Japan's Ministry of Health, Labor and Welfare. These designations are granted to therapies intended for the treatment of rare diseases, reflecting the critical need for innovative solutions for conditions like MSA, which affects fewer than 200,000 individuals in the United States.

Amlenetug is a human monoclonal antibody designed to target and bind to various forms of the protein α-synuclein, which is implicated in the pathology of MSA. By preventing the uptake and inhibiting the aggregation of this protein, amlenetug aims to modify the disease process. The drug's mechanism involves enhancing immune-mediated clearance of α-synuclein complexes through microglial uptake.

Lundbeck's development of amlenetug is part of a collaborative effort with Genmab A/S, under a joint research and licensing agreement. The company has initiated the MASCOT trial, a phase III study designed to further evaluate the efficacy, safety, and tolerability of amlenetug in MSA patients. The trial is being conducted in multiple regions, including North America, Europe, and Asia, and involves a randomized, double-blind, placebo-controlled design. Participants are assigned to receive either high or low doses of amlenetug or a placebo over 72 weeks, followed by an open-label extension period in which all participants can receive the active drug.

The recent AMULET trial, which laid the foundation for the Fast Track designation, involved a similar double-blind, placebo-controlled setup, assessing the impact of amlenetug on the clinical progression of MSA. The primary goal was to demonstrate a slowing of disease progression in patients who received the active treatment compared to those on placebo. Secondary objectives included evaluating the drug's effect on patients' functioning and disease severity. The trial showed promising results, with amlenetug administered intravenously every four weeks.

Multiple System Atrophy is a progressive disorder affecting the nervous system, characterized by the degeneration of nerve cells in the brain. Symptoms usually begin between the ages of 55 and 60, leading to a significant decline in patients' quality of life. MSA involves a range of symptoms, including issues with muscle control, balance, and bodily functions, often resembling those seen in Parkinson's disease. Currently, there is no cure or effective treatment to alter the progression of MSA, highlighting the urgent need for new therapeutic options like amlenetug.