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This medical policy (medical coverage guideline) is Copyright 2017, Blue Cross and Blue Shield of Florida (BCBSF). All Rights Reserved. You may not copy or use this document or disclose its contents without the express written permission of BCBSF. The medical codes referenced in this document may be proprietary and owned by others. BCBSF makes no claim of ownership of such codes. Our use of such codes in this document is for explanation and guidance and should not be construed as a license for their use by you. Before utilizing the codes, please be sure that to the extent required, you have secured any appropriate licenses for such use. Current Procedural Terminology (CPT) is copyright 2017 American Medical Association. All Rights Reserved. No fee schedules, basic units, relative values, or related listings are included in CPT. The AMA assumes no liability for the data contained herein. Applicable FARS/DFARS restrictions apply to government use. CPT® is a trademark of the American Medical Association. The use of specific product names is illustrative only. It is not intended to be a recommendation of one product over another, and is not intended to represent a complete listing of all products available.

09-J2000-13

Original Effective Date: 06/15/14

Reviewed: 05/14/14

Revised: 11/01/15

Subject: Elosulfase alfa (Vimizim™) Injection

THIS MEDICAL COVERAGE GUIDELINE IS NOT AN AUTHORIZATION, CERTIFICATION, EXPLANATION OF BENEFITS, OR A GUARANTEE OF PAYMENT, NOR DOES IT SUBSTITUTE FOR OR CONSTITUTE MEDICAL ADVICE. ALL MEDICAL DECISIONS ARE SOLELY THE RESPONSIBILITY OF THE PATIENT AND PHYSICIAN. BENEFITS ARE DETERMINED BY THE GROUP CONTRACT, MEMBER BENEFIT BOOKLET, AND/OR INDIVIDUAL SUBSCRIBER CERTIFICATE IN EFFECT AT THE TIME SERVICES WERE RENDERED. THIS MEDICAL COVERAGE GUIDELINE APPLIES TO ALL LINES OF BUSINESS UNLESS OTHERWISE NOTED IN THE PROGRAM EXCEPTIONS SECTION.

           
Dosage/ Administration Position Statement Billing/Coding Reimbursement Program Exceptions Definitions
           
Related Guidelines Other References Updates

 

           

DESCRIPTION:

Elosulfase alfa (Vimizim™) is the first agent approved by the US Food and Drug Administration (FDA) for the treatment of mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). Morquio A syndrome, which affects approximately 800 patients in the US, is characterized by the absence or marked reduction in N-acetylgalactosamine-6-sulfatase activity, resulting in the accumulation of the GAG substrates, KS and C6S, in the lysosomal compartment of cells throughout the body (accumulation leads to widespread cellular, tissue, and organ dysfunction). Elosulfase alfa is intended to provide the exogenous enzyme N-acetylgalactosamine-6-sulfatase that will be taken up into the lysosomes and increase the catabolism of the GAGs KS and C6S.

The safety and efficacy of elosulfase alfa were assessed in a 24-week, randomized, double-blind, placebo-controlled clinical trial of 176 patients with MPS IVA. The age of patients ranged from 5 to 57 years. Patients received elosulfase alfa 2 mg/kg once per week (n=58), 2 mg/kg once every other week (n=59), or placebo (n=59). The primary endpoint was the change from baseline in the distance walked in six minutes (6-MWT) at Week 24. The other endpoint included changes from baseline in the rate of stair climbing in three minutes (3-MSCT). The treatment effect in the 6-MWT, compared to placebo, was 22.5 m (95% CI: 4.0-40.9; p=0.0174) in patients who received elosulfase alfa 2 mg/kg once per week. There was no difference in 3-MSCT between treatment and placebo. Patients who received elosulfase alfa 2 mg/kg once every other week performed similarly to placebo.

POSITION STATEMENT:

Elosulfase alfa meets the definition of medical necessity when used for treatment of mucopolysaccharidosis type IVA (i.e., MPS IVA or Morquio A syndrome) and the prescribed dose is 2 mg/kg every week.

Approval duration: 1 year

DOSAGE/ADMINISTRATION:

THIS INFORMATION IS PROVIDED FOR INFORMATIONAL PURPOSES ONLY AND SHOULD NOT BE USED AS A SOURCE FOR MAKING PRESCRIBING OR OTHER MEDICAL DETERMINATIONS. PROVIDERS SHOULD REFER TO THE MANUFACTURER’S FULL PRESCRIBING INFORMATION FOR DOSAGE GUIDELINES AND OTHER INFORMATION RELATED TO THIS MEDICATION BEFORE MAKING ANY CLINICAL DECISIONS REGARDING ITS USAGE.

FDA-approved: elosulfase alfa is indicated for treatment of mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). The recommended dose is 2 mg/kg administered once weekly as an intravenous infusion over 3.5 to 4.5 hours.

Product Availability: elosulfase alfa is supplied as a 1 mg/mL single-use vial.

PRECAUTIONS:

Warnings:

  1. Anaphylaxis and Hypersensitivity Reactions: Life-threatening anaphylaxis and hypersensitivity reactions have been observed in some individuals during treatment with elosulfase alfa. If anaphylaxis or severe hypersensitivity reactions occur, immediately stop the infusion and initiate appropriate medical treatment. Pre-treatment with antihistamines with or without antipyretics is recommended prior to the start of infusion.
  2. Risk of Acute Respiratory Complications: Persons with acute febrile or respiratory illness may be at higher risk of life-threatening complications from hypersensitivity reactions. Careful consideration should be given to the patient's clinical status prior to administration of elosulfase alfa and consider delaying the elosulfase infusion.
  3. Pregnancy Category C: There is a Morquio A Registry that collects data on pregnant women with MPS IVA who are treated with Vimizim. Contact MARS@bmrn.com or call 1-800-983-4587 for information and enrollment

BILLING/CODING INFORMATION:

The following codes may be used to describe:

HCPCS Coding

J1322

Injection, elosulfase alfa, 1 mg

ICD-10 Diagnoses Codes That Support Medical Necessity (Effective 10/01/15)

E76.219

Morquio mucopolysaccharidoses, unspecified

E76.3

Mucopolysaccharidosis, unspecified

REIMBURSEMENT INFORMATION:

Refer to section entitled POSITION STATEMENT.

PROGRAM EXCEPTIONS:

Federal Employee Program (FEP): Follow FEP guidelines.

State Account Organization (SAO): Follow SAO guidelines.

Medicare Advantage Products: No National Coverage Determination (NCD) and/or Local Coverage Determination (LCD) were found at the time of the last guideline revised date.

Medicare Part D: Florida Blue has delegated to Prime Therapeutics authority to make coverage determinations for the Medicare Part D services referenced in this guideline.

DEFINITIONS:

Mucopolysaccharidosis IV Type A (i.e., MPS IV, Morquio A syndrome): a rare disorder in which the gene that encodes for N-acetylgalactosamine-6-sulfatase (GALNS) enzyme is mutated. This results in sub optimal GALNS activity that causes progressive accumulation of glycosaminoglycans in multiple organs and various tissues leading to significant morbidities and multisystemic clinical impairments resulting in diminished functional capacity, impaired quality of life, and early mortality.

RELATED GUIDELINES:

None.

OTHER:

None

REFERENCES:

1. Clinical Pharmacology [database online]. Tampa, FL: Gold Standard, Inc.;2012. URL www.clinicalpharmacilogy-ip.com Accessed 4/21/14

2. Ingenix HCPCS Level II, Expert 2014.

3. Ingenix ICD-9-CM for Physicians – Volumes 1 & 2, Expert 2014.

4. Kalydeco (ivacaftor) [package insert]. Vertex Pharmaceuticals, Inc. Cambridge (MA): August 2012February 2014.

5. Micromedex® Healthcare Series [Internet Database]. Greenwood Village, Colo: Thomson Healthcare. Updated periodically. Accessed 4/21/14.

6. Vimizim (elosulfase alfa) [package insert]. BioMarin Pharmaceutical Inc. Novartis (CA): February 2014.

COMMITTEE APPROVAL:

This Medical Coverage Guideline (MCG) was approved by the BCBSF Pharmacy Policy Committee on 05/14/14.

GUIDELINE UPDATE INFORMATION:

06/15/14

New Medical Coverage Guideline.

01/01/15

Revision to guideline; consisting of annual HCPCS coding update

11/01/15

Revision: ICD-9 Codes deleted.

Date Printed: August 23, 2017: 06:03 AM