Elosulfase alfa
- Generic Name
- Elosulfase alfa
- Brand Names
- Vimizim
- Drug Type
- Biotech
- CAS Number
- 9025-60-9
- Unique Ingredient Identifier
- ODJ69JZG85
- Background
Elosulfase alfa is a synthetic version of the enzyme N-acetylgalactosamine-6-sulfatase. It was approved by the FDA in 2014 for the treatment of Morquio syndrome. Elosulfase alfa was developed by BioMarin Pharmaceutical Inc. and is marketed under the brand Vimizim™. The recommended dose is 2 mg per kg given intravenously over a minimum range of 3.5 to 4.5 hours, based on infusion volume, once every week.
- Indication
Vimizim is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
- Associated Conditions
- Mucopolysaccharidosis Type IVA
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
- Conditions
- Mucopolysaccharidosis IV Type AMPS IVAMorquio A Syndrome
- First Posted Date
- 2014-11-19
- Last Posted Date
- 2024-03-05
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 418
- Registration Number
- NCT02294877
- Locations
- 🇺🇸
University of Southern California, Los Angeles, California, United States
🇺🇸Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois, United States
🇺🇸Arkansas Children's Hospital, Little Rock, Arkansas, United States
BMN 110 Phase 3B in Australian Patients
- First Posted Date
- 2013-10-21
- Last Posted Date
- 2019-09-26
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 13
- Registration Number
- NCT01966029
- Locations
- 🇦🇺
Princess Margaret Hospital for Children, Perth, Western Australia, Australia
🇦🇺Children's Hospital at Westmead, Westmead, New South Wales, Australia
🇦🇺Lady Cilento Children's Hospital (previous: Royal Children's Hospital), Brisbane, Queensland, Australia
BMN 110 US Expanded Access Program
- Conditions
- Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA
- First Posted Date
- 2013-05-21
- Last Posted Date
- 2014-04-02
- Lead Sponsor
- BioMarin Pharmaceutical
- Registration Number
- NCT01858103
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
- Conditions
- Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA
- Interventions
- First Posted Date
- 2012-10-02
- Last Posted Date
- 2016-01-12
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 16
- Registration Number
- NCT01697319
- Locations
- 🇬🇧
NIHR/Wellcome Trust Birmingham CRF, Queen Elizabeth Hospital, Birmingham, United Kingdom
🇩🇪Universitätsklinikum Hamburg, Hamburg, Germany
🇬🇧Salford Royal NHS Foundation Trust, Salford, United Kingdom
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
- Conditions
- Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA
- Interventions
- First Posted Date
- 2012-05-31
- Last Posted Date
- 2016-02-01
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 25
- Registration Number
- NCT01609062
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
- Conditions
- Morquio A SyndromeMPS IVAMucopolysaccharidosis IVA
- Interventions
- First Posted Date
- 2012-01-24
- Last Posted Date
- 2017-08-10
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 15
- Registration Number
- NCT01515956
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
- Conditions
- Mucopolysaccharidosis IVAMorquio A SyndromeMPS IV A
- Interventions
- First Posted Date
- 2010-11-16
- Last Posted Date
- 2015-09-30
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 20
- Registration Number
- NCT01242111
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
- First Posted Date
- 2009-04-21
- Last Posted Date
- 2014-06-30
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 20
- Registration Number
- NCT00884949
Drug Development Updates
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