Subject:
Pompe Disease Treatment with Lumizyme (Alglucosidase alfa)
Description:
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IMPORTANT NOTE:
The purpose of this policy is to provide general information applicable to the administration of health benefits that Horizon Blue Cross Blue Shield of New Jersey and Horizon Healthcare of New Jersey, Inc. (collectively “Horizon BCBSNJ”) insures or administers. If the member’s contract benefits differ from the medical policy, the contract prevails. Although a service, supply or procedure may be medically necessary, it may be subject to limitations and/or exclusions under a member’s benefit plan. If a service, supply or procedure is not covered and the member proceeds to obtain the service, supply or procedure, the member may be responsible for the cost. Decisions regarding treatment and treatment plans are the responsibility of the physician. This policy is not intended to direct the course of clinical care a physician provides to a member, and it does not replace a physician’s independent professional clinical judgment or duty to exercise special knowledge and skill in the treatment of Horizon BCBSNJ members. Horizon BCBSNJ is not responsible for, does not provide, and does not hold itself out as a provider of medical care. The physician remains responsible for the quality and type of health care services provided to a Horizon BCBSNJ member.
Horizon BCBSNJ medical policies do not constitute medical advice, authorization, certification, approval, explanation of benefits, offer of coverage, contract or guarantee of payment.
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Pompe disease (glycogen storage disease type II, GSD II, glycogenosis type II, or acid maltase deficiency) is an inherited disorder of glycogen metabolism caused by absence or marked deficiency of the lysosomal enzyme called acid alpha glucosidase (GAA). Pompe disease is a rare disorder that affects one in 40,000 to 300,000 individuals. In the infantile-onset form, which progresses rapidly, Pompe disease results in intralysosomal accumulation of glycogen in various tissues, particularly cardiac, skeletal, and hepatic tissues, leading to development of cardiomyopathy, progressive muscle weakness, and impairment of respiratory function. In the late-onset forms (juvenile and adult patients), which is correlated with slower progression, intralysosomal accumulation of glycogen is limited primarily to skeletal muscle, resulting in progressive muscle weakness. Adult onset disease usually presents as a slowly progressive proximal myopathy, with the variable addition of diaphragmatic and respiratory muscle paralysis. While juvenile and adult patients often die from respiratory failure, cardiorespiratory failure is the most common cause of death among infants.
Myozyme and Lumizyme (alglucosidase alfa) provides an exogenous source of GAA, exerting enzymatic activity in cleaving glycogen. Myozyme is the first therapy that was FDA approved for Pompe disease in April 2006 for use in patients with Pompe disease (GAA deficiency). Lumizyme (alglucosidase alfa) was approved in May 2010 for patients ages 8 years and older with late-onset (non-infantile) Pompe disease. Myozyme has been in short supply due to limited manufacturing capacity. Therefore, the manufacturer reserved Myozyme to treat infants and children with Pompe disease because younger patients generally have a much more aggressive form of the disease.
[INFORMATIONAL NOTE: Lumizyme and Myozyme are two different products, both manufactured by Genzyme Corp. In order to preserve the supply of Myozyme for infants and children who are restricted from using Lumizyme, it is necessary for all patients who are eligible for treatment with Lumizyme to receive therapy with this product. Based on the FDA-approved indication, the manufacturer recommends that Myozyme be reserved the following patient groups:
· patients under the age of 8 with either infantile-onset or late-onset Pompe disease
· patients of any age with a confirmed diagnosis of infantile-onset Pompe disease or evidence of cardiac hypertrophy (enlarged heart)
All patients who do not meet the above criteria should be transitioned to Lumizyme treatment.]
The FDA approved dosage for Myozyme and Lumizyme is 20 mg/kg body weight administered every 2 weeks as intravenous infusion. The total volume of infusion is determined by the patient’s body weight and should be administered over approximately 4 hours.
As of December 31, 2014, Myozyme is no longer available. The manufacturer of Myozyme has discontinued production based on the expanded Lumizyme label and has shifted all production resources to Lumizyme. Patients that have received Myozyme are encouraged to discuss the need to transition from Myozyme to Lumizyme with their doctor as necessary. For patients treated with Myozyme regardless of age and phenotype, Lumizyme is available.
[INFORMATIONAL NOTE: According to the package insert, it is recommended that patients be monitored for IgG antibody formation every 3 months. To ensure patient safety, Genzyme has established a comprehensive Risk Management Plan for alglucosidase alfa, which has identified product-and disease-related risks, and provides a framework for mitigation of these risks.]
[INFORMATIONAL NOTE: The product labeling for Lumizyme contains the following BLACK BOX WARNINGS:
- Life-threatening anaphylactic reactions, severe allergic reactions and immune mediated reactions have been observed in some patients during Lumizyme infusions. Therefore, appropriate medical support should be readily available when Lumizyme is administered.
- Because of the potential risk of rapid disease progression in Pompe disease patients less than 8 years of age, Lumizyme is available only through a restricted distribution program called the Lumizyme ACE Program. Only prescribers and healthcare facilities enrolled in the program may prescribe, dispense or administer Lumizyme. Lumizyme may be administered only to patients who are enrolled in and meet all the conditions of the Lumizyme ACE Program.]
Policy:
(NOTE: For Medicare Advantage, please refer to the Medicare Coverage Section below for coverage guidance.)
The requirements of the Horizon BCBSNJ Pompe Disease Treatment with Lumizyme (Alglucosidase alfa) Program may require a precertification/prior authorization via MagellanRx Management. These requirements are member-specific: please verify member eligibility and requirements through the Horizon Provider Portal (www.horizonblue.com/provider). Ordering clinicians should request pre-certification from MagellanRx Management at ih.magellanrx.com or call 1-800-424-4508 (when applicable).
I. Please refer to a separate policy on Site of Administration for Infusion and Injectable Prescription Medications (Policy #142) under the Drug Section.
II. Alglucosidase alfa (Lumizyme)
1. Alglucosidase alfa (Lumizyme) is medically necessary when one of the following criteria are met:
a. Members receiving Lumizyme must have a diagnosis of Pompe disease (infantile-onset or late-onset),
i. For infantile-onset Pompe disease, members must meet the following (documentation of medical records required):
1. Documented baseline values for one or more of the following: muscle weakness, motor function, respiratory function, cardiac involvement, percent predicted forced vital capacity (FVC), and/or 6 minute walk test (6MWT), OR
ii. For late-onset Pompe disease, members must meet the following (documentation of medical records required):
1. Documented baseline levels for FVC or 6MWT; AND
b. Diagnosis of Pompe disease is documented by a deficiency of acid alpha-glucosidase (GAA) enzyme activity by a GAA enzyme assay (documentation of medical records required); OR
c. Detection of pathogenic variants in the GAA gene by molecular genetic testing (documentation of medical records required).
[INFORMATIONAL NOTE: Clinical signs of infantile-onset Pompe disease include hypotonia, severe cardiomegaly, respiratory infections, failure to reach motor milestones, and difficulty feeding. Clinical signs of late-onset Pompe disease include progressive proximal muscle weakness and respiratory insufficiency, gait abnormalities, creatinine kinase levels normal to elevated, arrhythmias, and feeding and swallowing difficulties.
According to the FDA-approved product information, Lumizyme is indicated for use in patients with Pompe disease. The ADVANCE trial of patients 12 months and older previously treated with Myozyme and switched to Lumizyme as part of the trial demonstrated that alglucosidase alfa 4000L (Lumizyme) is comparable to alglucosidase alfa 160L (Myozyme®). Lumizyme is now indicated for patients with Pompe disease. There is no limitation as to age and phenotype.
Based on the product information for Lumizyme, Infantile-onset Pompe disease patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to fluid overload, and require additional monitoring. Life-threatening anaphylaxis and hypersensitivity reactions have been observed in some patients during and after treatment with alglucosidase alfa.]
2. When medically necessary, coverage for the initial dosage and renewal dosage of alglucosidase alfa (Lumizyme) approval will be based on the FDA-recommended dosing of 20 mg/kg body weight every 2 weeks.
[INFORMATIONAL NOTE: The only FDA-recommended dosing of Lumizyme is 20mg/kg body weight every 2 weeks. However, a number of case reports and one small study have suggested the efficacy of higher doses. The majority of data supports use of up to 40mg/kg every other week although one small study has suggested possible benefit may be seen with the use of 40mg/kg every week as well.]
3. Continued therapy with alglucosidase alfa (Lumizyme) will be considered annually based on the following criteria:
a. Absence of unacceptable toxicity, such as severe cutaneous and systemic immune mediated reactions, acute cardiorespiratory failure, severe allergic and anaphylactic reactions, cardiac arrhythmia and sudden cardiac death during general anesthesia, antibody development, etc. AND
b. No evidence that patient has developed IgG antibodies to algucosidase alfa a sustained titer level of ≥ 12,800, AND
c. Patient has demonstrated a beneficial response to therapy compared to pretreatment baseline in one or more of the following (documentation of medical records required):
a. For infantile-onset diseae:
i. Stabilization or improvement in muscle weakness, motor function, respiratory function, cardiac involvement, FVC, and/or 6MWT
b. For late-onset disease: stabilization or improvement in FVC and/or 6MWT
4. Alglucosidase alfa (Lumizyme) is considered investigational for all other conditions.
Medicare Coverage
There is no National Coverage Determination (NCD or Local Coverage Determination (LCD) for jurisdiction JL for this service. Therefore, Medicare Advantage Products will follow the Horizon BCBSNJ Medical Policy.
**Note: Bullet 1 of the policy section referring to Site of Administration for Infusion and Injectable Prescription Medications (Policy #142) does not apply for Medicare Advantage Products.
Medicaid Coverage
For Horizon NJ Health members, please follow this link for the corresponding HNJH drug policy https://services3.horizon-bcbsnj.com/ddn/NJhealthWeb.nsf
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Horizon BCBSNJ Medical Policy Development Process:
This Horizon BCBSNJ Medical Policy (the “Medical Policy”) has been developed by Horizon BCBSNJ’s Medical Policy Committee (the “Committee”) consistent with generally accepted standards of medical practice, and reflects Horizon BCBSNJ’s view of the subject health care services, supplies or procedures, and in what circumstances they are deemed to be medically necessary or experimental/ investigational in nature. This Medical Policy also considers whether and to what degree the subject health care services, supplies or procedures are clinically appropriate, in terms of type, frequency, extent, site and duration and if they are considered effective for the illnesses, injuries or diseases discussed. Where relevant, this Medical Policy considers whether the subject health care services, supplies or procedures are being requested primarily for the convenience of the covered person or the health care provider. It may also consider whether the services, supplies or procedures are more costly than an alternative service or sequence of services, supplies or procedures that are at least as likely to produce equivalent therapeutic or diagnostic results as to the diagnosis or treatment of the relevant illness, injury or disease. In reaching its conclusion regarding what it considers to be the generally accepted standards of medical practice, the Committee reviews and considers the following: all credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, physician and health care provider specialty society recommendations, the views of physicians and health care providers practicing in relevant clinical areas (including, but not limited to, the prevailing opinion within the appropriate specialty) and any other relevant factor as determined by applicable State and Federal laws and regulations.
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Index:
Pompe Disease Treatment with Lumizyme (Alglucosidase alfa)
Myozyme (Alglucosidase alfa)
Lumizyme (Alglucosidase alfa)
Alglucosidase alfa
References:
1. Product Information: Myozyme (alglucosidase alfa). Genzyme Corporation, Cambridge, MA. May 2019.
2. ClinicalTrials.gov. Alglucosidase alfa. [2/18/2014]. Available at: www.clinicaltrials.gov.
3. New York Times: Business. Pollack A. Genzyme’s Drug for Rare Enzyme Deficiency is Approved. April 29, 2006. Available at: http://www.nytimes.com/2006/04/29/business/29drug.html?ex=1150344000&en=4792984793db634d&ei=5070
4. U.S. Food and Drug Administration. FDA News: FDA Approves First Treatment for Pompe Disease. April 28, 2006. Available at: http://www.fda.gov/bbs/topics/NEWS/2006/NEW01365.html
5. Kishnani P, ByrneB, Nicolino M, et al. Enzyme replacement therapy (ERT) with recombinant human acid alpha glucosidase (RHGAA) in infantile onset Pompe disease (IOPD) [abstract]. J Inherit Metab Dis 2005; 28: 195.
6. Kishnani P, Byrne B, Nicolino M, et al. Enzyme replacement therapy (ERT) with recombinant human acid alpha glucosidase (rhGAA) in infantile-onset Pompe disease: interim results from a pivotal trial [abstract]. 55th Annual Meeting of the American Society of Human Genetics; October 25-29; Salt Lake City, Utah; 2005. p. 199.
7. Nicolino M, Kishnani P, Spencer C, et al. Safety and efficacy of cho-cell derived recombinant human acid alpha glucosidase (rhGAA) in patients with infantile-onset Pompe disease (IOPD) treated after 6 months of age [abstract]. Second Annual Symposium on Lysosomal Storage Disorders; March 2-4; Athens, Greece; 2005. p. 31-32.
8. Kishnani P, Spencer C, Nicolino M, et al. Safety and efficacy of CHO-cell derived recombinant human acid alpha glucosidase (rhGAA) in patients with infantile-onset Pompe disease (IOPD) treated after 6 months of age [abstract]. Annual Clinical Genetics Meeting, American College of Medical Genetics; March 17-20; Dallas, TX; 2005. p. 55.
9. Kishnani P, Spencer C, Byrne B, et al. Long-term efficacy of enzyme replacement therapy (ERT) in children with Pompe disease [abstract]. Annual Clinical Genetic Meeting, American College of Medical Genetics; Mar 23-26; San Diego, CA; 2006.
10. van der Ploeg A, Reuser A, Van Cappelle C. Early signs of benefit in children with late- onset Pompe disease following the first 6 months of treatment with enzyme replacement therapy (ERT) with recombinant human acid alpha glucosidase (rhGAA) in an open label study [abstract]. Annual Clinical Genetic Meeting, American College of Medical Genetics; Mar 23-26; San Diego, CA; 2006.
11. Worden M, Morgan C, Kingma W. Safety monitoring and risk management of alglucosidase alfa for Pompe disease [abstract]. Presented at the Steps Forward in Pompe Disease International Symposium; Apr 5-6, 2006; Berlin, Germany. P. 58-9.
12. Van Capelle C, Hagemans M, et al. 6.5 years follow-up of 3 patients with late-onset Pompe disease treated with recombinant human a-glucosidase [abstract]. Presented at the International Workshop on Lysosomal Storage Disorders; Nov 10-11, 2006; Budapest, Hungary. P. 36.
13. Van der Ploeg A, Reuser A, Van Capelle C. Early signs of benefit in children with late-onset Pompe disease following the first 6 months of treatment with enzyme replacement therapy (recombinant human acid alpha-glucosidase) in an open-label study [abstract]. Neuromuscul Disord 2006; 16 Suppl 1:S183.
14. Deegan P, Waldek, S, et al. Guidelines for the investigation and management of late onset acid maltase deficiency (type II glycogen storage disease/Pompe disease). Aug 3, 2007.
15. Wraith, JE, Lee, P, et al. Guidelines for the investigation and management of infantile Pompe disease. Aug 2007.
16. MediLexicon. Myozyme (alglucosidase alfa). August 2008. Available at: http://www.medilexicon.com/drugs/myozyme.php.
17. A physician’s guide to Pompe disease. Genzyme Corporation, Cambridge, MA. 2005. Available at: http://worldpompe.org/images/uploads/pompe_physiciansguide.pdf.
18. Genzyme study of myozyme for late-onset Pompe patients meets co-primary efficacy endpoints. December 13, 2007. Available at: http://www.genzyme.com/corp/media/GENZ%20PR-121307.asp.
19. Scientific discussion of Myozyme. The European Medicines Agency (EMEA) 2006. Available at: http://www.emea.europa.eu/humandocs/PDFs/EPAR/myozyme/H-636-en6.pdf.
20. Clinical Pharmacology [database online]. Alglucosidase alfa. Gold Standard; 2009. Updated May 8, 2009.
21. FDA Approves New Treatment for Late-Onset Pompe Disease. FDA News Release. May 25, 2010. Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm213282.htm.
22. Lumizyme (alglucosidase alfa) Prescribing Information. Genzyme Corporation, Cambridge, MA. 2014
23. Van der Ploeg AT, Clemens PR, Corzo D, et al. A randomized study of alglucosidase alfa in late-onset Pompe's disease. N Engl J Med. 2010;362(15):1396-406.
24. Strothotte S, Strigl-Pill N, Grunert B, et al. Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial. J Neurol. 2010;257(1):91-7.
25. Kishnani PS, Corzo D, Leslie ND, et al. Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease. Pediatr Res. 2009;66(3):329-35.
26. United States Pompe Program Update, May 25, 2010. Genzyme.
27. MICROMEDEX® 2.0 (Healthcare Series). DRUGDEX® Evaluations. Alglucosidase alfa. Available at: http://www.micromedexsolutions.com. Accessed February 25, 2015.
28. Clinical Pharmacology [database online]. Alglucosidase alfa. Gold Standard; 2009. Updated August 06, 2014. Accessed March 09, 2015.
29. Alglucosidase alfa. In: McEvoy GK, editor. AHFS: Drug Information (2015). Bethesda, MD: American Society of Health-System Pharmacists; 2015. Updated October 30, 2014. Accessed March 09, 2015. http://online.statref.com/Document.aspx?fxId=1&docId=972
30. Mattosova S, Hlavata A, Spalek P, et al. Diagnostic Algorithm Late onset form of Pompe disease. Bratisl Med J 2015;116(8):502-5.
31. Van Gelder CM, Poelman E, Plug I, et al. A higher dose of enzyme therapy in patients with classic infantile Pompe disease seems to improve ventilator-free survival and motor function. Poster presentation abstract published in BMC Musculoskeletal Disorders. 2013, 14 (Suppl 2): P19.
32. Case LE, Bjartmar C, Morgan C, et al. Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease. Neuromuscular Disorders 25 (2015) 321-332.
33. Markic J, Polic B, Kuzmanic-Samija R, et al. Immune Modulation Therapy in CRIM-Positive and IgG Antibody-Positive Infant with pompe Disease Treated with Alglucosidase Alfa: A Case Report. JIMD Rep. 2012; 2: 11-15.
34. Yanovitch et al. Improvement of Bilateral Ptosis on Higher Dose Enzyme Replacement Therapy in Pompe Disease. J Neuro-Ophthalmol 2010; 30: 165-166.
35. Case LE, Beckemeyer AA, Kishnani PS. Infantile Pompe Disease on ERT- Update on Clinical Presentation, Musculoskeletal Management, and Exercise Consideration. Am J Med Genet Part C Semin Med Genet 160C: 69-79.
36. Hirschhorn, Rochelle and Arnold J. J. Reuser. Glycogen Storage Disease Type II: Acid Alpha-glucosidase (Acid Maltase) Deficiency. In: Scriver C, Beaudet A, Sly W, Valle D, editors. The Metabolic and Molecular Bases of Inherited Disease. 8th Edition. New York: McGraw-Hill, 2001. 3389-3420.
37. Kishnani PS, Howell RR. Pompe disease in infants and children. J Pediatr 2004;144:S35-43.
38. Genzyme: Press Release. United States Pompe Community Update - October 17, 2014. Available at: amda-pompe.org/downloads/news/2014-10-17_US_Pompe_Community_Update_MZ.pdf. Accessed June 2018.
Codes:
(The list of codes is not intended to be all-inclusive and is included below for informational purposes only. Inclusion or exclusion of a procedure, diagnosis, drug or device code(s) does not constitute or imply authorization, certification, approval, offer of coverage or guarantee of payment.)
CPT*
HCPCS
J0221
* CPT only copyright 2020 American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.
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Medical policies can be highly technical and are designed for use by the Horizon BCBSNJ professional staff in making coverage determinations. Members referring to this policy should discuss it with their treating physician, and should refer to their specific benefit plan for the terms, conditions, limitations and exclusions of their coverage.
The Horizon BCBSNJ Medical Policy Manual is proprietary. It is to be used only as authorized by Horizon BCBSNJ and its affiliates. The contents of this Medical Policy are not to be copied, reproduced or circulated to other parties without the express written consent of Horizon BCBSNJ. The contents of this Medical Policy may be updated or changed without notice, unless otherwise required by law and/or regulation. However, benefit determinations are made in the context of medical policies existing at the time of the decision and are not subject to later revision as the result of a change in medical policy
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