China’s Wanbangde New Building Materials Co., Ltd (SHE: 002082) announced that its mecobalamin product has received an orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS). The designation highlights the drug’s potential to address unmet medical needs in ALS, a neurodegenerative disorder with limited therapeutic options.
Drug Profile
Mecobalamin, a coenzyme of methionine synthase, plays a critical role in maintaining the methylation cycle by converting homocysteine to methionine. Elevated homocysteine levels in ALS patients’ cerebrospinal fluid are linked to neurotoxicity. Mecobalamin reduces homocysteine accumulation through metabolic regulation and directly protects neurons from glutamate toxicity, promoting nerve regeneration and repair in animal models of sciatic nerve injury and diabetic neuropathy.
Clinical Evidence
Clinical studies show mecobalamin’s dual neuroprotective mechanism: lowering homocysteine levels and shielding neurons from excitotoxicity. In animal trials, the drug improved nerve conduction function in models of peripheral neuropathy, underscoring its potential in neurodegenerative disease treatment.
Regulatory Status
Mecobalamin freeze-dried powder injection was approved in Japan for ALS treatment in September 2022. While no mecobalamin product is approved in the U.S. yet, the FDA’s orphan drug designation provides Wanbangde incentives to advance development, including tax credits and exclusive marketing rights upon approval.-Fineline Info & Tech
