Tralesinidase alfa - BioMarin Pharmaceutical
Alternative Names: Alpha-N-acetyglucosaminidase-insulin-like-growth-factor-2 fusion protein; AX-250; BMN 250; NAGLU-IGF2 fusion protein; TA-ERT - BioMarin PharmaceuticalLatest Information Update: 15 May 2025
At a glance
- Originator BioMarin Pharmaceutical
- Class Acetylglucosaminidases; Recombinant fusion proteins
- Mechanism of Action Alpha N acetyl D glucosaminidase replacements
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Phase I/II Mucopolysaccharidosis III
Most Recent Events
- 07 May 2025 Spruce plans to launch Tralesinidase alfa in Latin America, Turkey, Asia, and other international markets
- 06 May 2025 Tralesinidase alfa - BioMarin Pharmaceutical receives Fast Track designation for Mucopolysaccharidosis III [Intracerebral] (In children, In infants) in USA prior to May 2025
- 06 May 2025 Tralesinidase alfa - BioMarin Pharmaceutical receives Priority Medicine (PRIME) status for Mucopolysaccharidosis III in European Union prior to May 2025