Elosulfase alfa

Overview

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

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)	2014/11/19	NCT02294877	N/A	Completed	BioMarin Pharmaceutical
BMN 110 Phase 3B in Australian Patients	2013/10/21	NCT01966029	Phase 3	Completed	BioMarin Pharmaceutical
BMN 110 US Expanded Access Program	2013/05/21	NCT01858103	N/A	APPROVED_FOR_MARKETING	BioMarin Pharmaceutical
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation	2012/10/02	NCT01697319	Phase 2	Terminated	BioMarin Pharmaceutical
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome	2012/05/31	NCT01609062	Phase 2	Terminated	BioMarin Pharmaceutical
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)	2012/01/24	NCT01515956	Phase 2	Completed	BioMarin Pharmaceutical
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)	2010/11/16	NCT01242111	Phase 1	Terminated	BioMarin Pharmaceutical
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA	2009/04/21	NCT00884949	Phase 1	Completed	BioMarin Pharmaceutical

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
VIMIZIM	BioMarin Pharmaceutical Inc.	68135-100	INTRAVENOUS	5 mg in 5 mL	1/21/2021

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Vimizim	BioMarin International Limited,Shanbally,Ringaskiddy,County Cork,P43 R298,Ireland	Authorised	4/27/2014

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
VIMIZIM CONCENTRATE FOR SOLUTION FOR INFUSION 1MG/ML	N/A	BIOMARIN PHARMACEUTICAL INC.	N/A	N/A	6/26/2015

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
VIMIZIM elosulfase alfa (rch) 1 mg/mL concentrated solution for injection vial	215523	BioMarin Pharmaceutical Australia Pty Ltd	Medicine	A	12/9/2014

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
VIMIZIM	Biomarin International Limited	02427184	Solution - Intravenous	1 MG / ML	8/27/2014

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
No CIMA AEMPS (Spain) approvals found for this drug.

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

Related News

NICE Provisionally Approves BioMarin's Vimizim for Ultra-Rare Morquio Disease with Real-World Data Requirements

10 years ago

• NICE has issued a provisional approval for BioMarin's Vimizim (elosulfase alfa), priced at £395,000 per patient annually, for treating the ultra-rare Morquio disease affecting 88 patients in England. • The approval is contingent on implementing real-world data collection and establishing a price cap, marking NICE's innovative approach to managing high-cost rare disease treatments. • The MPS Society welcomes the decision while expressing concerns about further delays in the approval process, as patients urgently await access to this life-changing therapy.

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