Prior Auth Protocol

FDA Approved Indications:

For patients with Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome).

Health Net Approved Indications and Usage Guidelines:

Diagnosis of Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome).

Coverage is Not Authorized For:

Non-FDA approved indications, which are not listed in the Health Net Approved Indications and Usage Guidelines section, unless there is sufficient documentation of efficacy and safety in the published literature.

General Information:

MPS VI is an autosomal recessive lysosomal storage disorder characterized by a deficiency of N-acetylgalactosamine 4-sulfatase. This deficiency results in accumulation of a glycosaminoglycan (GAG) substrate (dermatan sulfate) throughout the body, leading to widespread and progressive cellular, tissue, and organ dysfunction.
In a study involving 39 patients, the observed mean (SD) walk distance for the Naglazyme group at baseline was 227 (170) m for the 12 minute walk test (12MWT). After 24 weeks, patients receiving Naglazyme walked on average 92 meters (41%) more in the 12MWT (p=0.025). The Urinary glycosaminoglycan (GAG) declined by mean 227 (18) microgram/mg more with Naglazyme than placebo (75%) (p<0.001).
Fifty-six patients derived from 3 clinical studies were followed in open-label extension studies for 97-260 weeks. All patients received weekly infusions of Naglazyme at 1 mg/kg. The average improvement in the 6-minute walk test (6MWT) ranged from 29% to 63%. A significant reduction of 71-79% in urinary GAG was sustained.

Therapeutic Alternatives:

Drug	Dosing Regimen	Dose Limit/ Maximum Dose
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* Requires Prior Authorization

Recommended Dosing Regimen and Authorization Limit:

Drug	Dosing Regimen	Authorization Limit
Naglazyme	1 mg per kg of body weight administered once weekly as an IV infusion	Medical: 6 months or renewal date, whichever is longer.

Product Availability:

Vial: 5 mg per 5 mL

References:

Naglazyme [prescribing information]. Novato, CA: BioMarin; March 2013.
Giugliani R, Harmatz P, Wraith JE. Management guidelines for mucopolysaccharidosis VI. Pediatrics. 2007 Aug;120: 405-18.
Harmatz P, Giugliani R, Schwartz IV, Guffon N, Teles EL, et al. Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Mol Genet Metab. 2008;94:469-75.
Harmatz P, Giugliani R, Schwartz I, Guffon N, Teles EL, et al. Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr. 2006;148:533-539.
Clinical Pharmacology Web site. Available at: http://clinicalpharmacology-ip.com/. Accessed June 17, 2015.
Naglazyme. American Hospital Formulary Service Drug Information. Available at: http://www.medicinescomplete.com/mc/ahfs/current/. Accessed June 17, 2015.
Galsulfase. In: DrugPoints^R System [Internet database]. Greenwood Village, Colo: Thomson Healthcare. Updated periodically. Accessed June 17, 2015.
Giugliani R, Lampe C, Harmatz P, etal. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)-10-year follow-up of patients who previously participated in an MPS VI survey study. AJMG 2014 Aug;164A(8):1953-64. Accessed at: http://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.36584/abstract;jsessionid=6460F8217ACFC3A1F505AFB9602442FB.f02t01