Denmark-based pharmaceutical company Lundbeck A/S (OTCMKTS: HLUBF) announced receiving Orphan Drug Designation (ODD) from Japan’s Ministry of Health, Labor, and Welfare (MHLW) for its investigational drug amlenetug. This designation positions amlenetug as a promising new treatment option for patients with multiple system atrophy (MSA), a rare and progressive neurodegenerative disorder.
Mechanism of Action
Amlenetug, a human monoclonal antibody (mAb) co-developed by Genmab A/S, targets all major forms of extracellular α-synuclein. By binding to this protein, amlenetug aims to prevent its uptake and inhibit the seeding of aggregation, addressing a key pathological mechanism in MSA.
Global Recognition and Previous Designations
Amlenetug has previously received Orphan Drug Designations in the US and European Union in April 2024 and May 2021, respectively. It also obtained SAKIGAKE designation from the MHLW in March 2023 and Fast Track Designation from the US FDA in February 2024. These recognitions highlight the potential of amlenetug to address significant unmet needs in the treatment of MSA.
Implications for Patients
The receipt of ODD in Japan marks another milestone in the development of amlenetug, bringing the company closer to providing a novel therapeutic option for MSA patients. The designation may also facilitate faster regulatory review and market access in Japan, potentially improving outcomes for those affected by this debilitating condition.-Fineline Info & Tech
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