Lundbeck’s Amlenetug receives FDA fast-track for neurodegenerative disorder

USA—The US Food and Drug Administration (FDA) has granted Lundbeck fast-track designation for its investigational drug, amlenetug.

This designation is expected to accelerate the review process for the drug, which is being developed as a potential treatment for multiple system atrophy (MSA), a serious and progressive neurodegenerative disorder.

The fast-track status means that amlenetug will benefit from more frequent interactions with the FDA and the possibility of rolling data submissions.

Now, Lundbeck may be able to bring this promising treatment to patients faster than under the usual review process.

This decision was largely influenced by the encouraging outcomes observed in the Phase II AMULET trial, which showed potential benefits in addressing the challenging symptoms of MSA.

Johan Luthman, Lundbeck’s head of research and development and executive vice-president, expressed his satisfaction with the FDA’s decision.

 He explained that obtaining fast-track designation is not only a recognition of the drug’s promise but also a vital step toward meeting the significant unmet needs of patients suffering from this debilitating condition.

His comments reflect the company’s commitment to accelerating the development of therapies that could make a real difference in people’s lives.

Furthermore, amlenetug has already achieved several regulatory milestones.

Prior to the fast-track designation, it received orphan drug status in the United States and from the European Medicines Agency back in May 2021.

Additionally, in March 2023, Japan’s Ministry of Health, Labour and Welfare awarded the drug the SAKIGAKE, or “pathfinder,” designation.

These recognitions highlight the drug’s potential and the global urgency for new treatments in neurodegenerative diseases.

At its core, amlenetug is a human monoclonal antibody engineered to target and bind to extracellular α-synuclein, a protein that plays a key role in the progression of MSA.

By attaching to this protein, the drug aims to block its uptake by cells and prevent the formation of harmful aggregates.

Moreover, the active Fc region of amlenetug might stimulate the immune system’s microglia to clear away α-synuclein, further reducing its harmful effects.

The development of amlenetug is being carried out under a joint research and licensing agreement between Lundbeck and Genmab.

The drug is now in a Phase III trial, known as the MASCOT study, which is a randomized, double-blind, placebo-controlled trial with an optional open-label extension.

This large-scale study, which spans Europe, North America, and Asia, is designed to thoroughly assess the safety and effectiveness of amlenetug in patients with MSA.

In addition to these scientific advancements, Lundbeck has strategically expanded its footprint in the neuro-rare disease sector by acquiring Longboard Pharmaceuticals in December 2024.

This acquisition, which secured all outstanding shares of Longboard, reinforces Lundbeck’s position as a leader in developing innovative treatments for rare neurological conditions.

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