Lundbeck has gained orphan drug designation (ODD) from Japan’s Ministry of Health, Labor and Welfare (MHLW) for amlenetug to treat multiple system atrophy (MSA).
The designation adds to the company’s previous recognitions: Japan’s SAKIGAKE designation and ODDs from the US Food and Drug Administration and the European Medicines Agency.
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Amlenetug is tailored to attach to all major extracellular α-synuclein forms.
The human monoclonal antibody aims to prevent the uptake and block aggregation seeding. Its active Fc region may also facilitate an increase in immune-mediated clearance of α-synuclein/mAb complexes via microglial uptake.
The development of the antibody is the result of a joint research and licensing agreement between the company and Danish biotechnology firm Genmab.
Lundbeck has commenced a randomised, parallel-group Phase III trial named MASCOT to evaluate the safety and efficacy of the antibody in treating MSA.
The trial will be conducted across Asia, Australia, Europe and North America. It consists of two segments.
Subjects in the double-blind period will be given high or low doses of amlenetug, or a placebo, for 72 weeks, followed by an open-label period during which all will be offered the antibody treatment.
The primary goal of the trial is to assess the antibody’s safety, efficacy and tolerability. It will be administered as an intravenous infusion every four weeks.
Lundbeck research and development head and executive vice-president Johan Luthman stated: “We are pleased to receive ODD for amlenetug in Japan. This designation highlights the potential of amlenetug as a treatment option for people living with MSA.
“We are hopeful of the potential for amlenetug to slow the clinical progression of this devastating disease and look forward to advancing its development through the MASCOT trial.”
In a move to strengthen its presence in the neuro-rare disease sector, Lundbeck completed the acquisition of all outstanding shares of Longboard Pharmaceuticals in December 2024.
