Drug intelligence / Profile preview

Rozebalamin (methylcobalamin)

Development stage
Phase 3
Lead developer
Eisai
Modality
Small Molecules
Administration
Intramuscular
01

Overview

Rozebalamin is an ultra-high-dose formulation of methylcobalamin, the biologically active form of vitamin B12, developed by Eisai for the treatment of amyotrophic lateral sclerosis (ALS). Approved in Japan in September 2024, it is administered via intramuscular injection at a dose of 50 mg twice weekly. While low-dose methylcobalamin is traditionally used for peripheral neuropathies and anemia, this ultra-high-dose regimen is designed to slow functional decline in ALS patients, particularly those in the early stages of the disease. Its mechanism of action is thought to involve the reduction of neurotoxic homocysteine levels and the mitigation of oxidative stress, glutamate toxicity, and neuronal apoptosis.

Brand names
Rozebalamin
Other names
mecobalaminVitamin B12
02

Targets

MTR (Methionine synthase)

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