Lundbeck’s Amlenetug receives Orphan Drug Designation in Japan for MSA

JAPAN—Lundbeck’s investigational drug, amlenetug, has been granted Orphan Drug Designation (ODD) by Japan’s Ministry of Health, Labor, and Welfare (MHLW) for patients with Multiple System Atrophy (MSA),

This designation highlights the potential of amlenetug as a promising treatment option for MSA, a rare and progressive neurological disorder that currently lacks approved therapies.

Amlenetug is a human monoclonal antibody designed to target and bind to all major forms of extracellular α-synuclein, a protein implicated in the progression of MSA.

By preventing the uptake and inhibiting the aggregation of α-synuclein, amlenetug aims to slow the clinical progression of this debilitating disease.

Additionally, its active Fc region may enhance immune-mediated clearance of α-synuclein/antibody complexes through microglia-mediated uptake, further supporting its therapeutic potential.

This recent designation in Japan adds to a series of significant recognitions amlenetug has received.

Amlenetug previously received the SAKIGAKE designation in Japan in March 2023, the Orphan Drug Designation from the U.S. FDA in April 2024, and the Orphan Drug Designation from the European Medicines Agency (EMA) in May 2021.

These designations not only highlight the drug’s potential but also facilitate its development by providing regulatory benefits such as expedited review processes.

Johan Luthman, Executive Vice President and Head of Research and development at Lundbeck, expressed his excitement about the new designation, highlighting its emphasis on amlenetug’s ability to meet the considerable unmet needs in treating MSA.

He noted that Lundbeck is hopeful that amlenetug can effectively slow the clinical progression of this devastating disease and is committed to advancing its development through ongoing clinical trials.

Lundbeck has recently initiated the MASCOT trial, a phase III clinical study designed to evaluate the efficacy, safety, and tolerability of amlenetug in patients with MSA.

 This trial is a randomized, double-blind, placebo-controlled study that will be conducted globally across North America, Europe, Asia, and Australia.

The trial consists of two parts: a double-blind period where participants receive either high or low doses of amlenetug or a placebo for 72 weeks, followed by an open-label extension period where all participants will be offered treatment with amlenetug.

The MASCOT trial builds on the promising results from the phase II AMULET trial, which showed a trend towards slowing clinical progression in patients with MSA.

Although the primary endpoint was not met statistically, the trial demonstrated a non-statistically significant slowing of disease progression by 19% as measured by the Unified Multiple System Atrophy Rating Scale (UMSARS).

Notably, in less impaired patients, amlenetug resulted in a more pronounced slowing of clinical progression by 37%.

Amlenetug will be administered as an intravenous infusion every four weeks, ensuring consistent and controlled drug delivery.

The initiation of the MASCOT trial marks a crucial step forward in Lundbeck’s efforts to develop a disease-modifying treatment for MSA, offering new hope for patients and families affected by this condition.

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