Drug Profile

Research programme: glycan and glycolipid synthesis inhibitors - BioMarin Pharmaceutical

Latest Information Update: 09 Apr 2018

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At a glance

Originator Zacharon Pharmaceuticals
Developer BioMarin Pharmaceutical
Class Small molecules
Mechanism of Action Ganglioside inhibitors; Glycosaminoglycan inhibitors; Polysaccharide inhibitors

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

Discontinued Brain cancer; Gangliosidoses; Mucopolysaccharidosis I; Mucopolysaccharidosis II; Mucopolysaccharidosis III

04 Nov 2017 No recent reports of development identified for preclinical development in Gangliosidoses in USA
04 Nov 2017 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-I in USA
04 Nov 2017 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-II in USA

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