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5.01.570 Pharmacologic Treatment of Duchenne Muscular Dystrophy

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5.01.570 Pharmacologic Treatment of Duchenne Muscular Dystrophy PHARMACY / MEDICAL POLICY – 5.01.570 Pharmacologic Treatment of Duchenne Muscular Dystrophy Effective Date: Aug. 6, 2021 RELATED MEDICAL POLICIES: Last Revised: June 11, 2021 11.01.523 Site of Service: Infusion Drugs and Biologic Agents Replaces: N/A Select a hyperlink below to be directed to that section. POLICY CRITERIA | DOCUMENTATION REQUIREMENTS | CODING RELATED INFORMATION | EVIDENCE REVIEW | REFERENCES | HISTORY ∞ Clicking this icon returns you to the hyperlinks menu above. Introduction Amondys 45® (casimersen), Exondys 51® (eteplirsen), Viltepso™ (vitolarsen), and Vyondys 53® (golodirsen) are new drugs used for Duchenne Muscular Dystrophy (DMD), which is a rare genetic disorder characterized by progressive muscle degeneration and weakness. It is caused by an absence of a functional dystrophin, which is a protein that helps keep muscle cells intact. Dystrophin gene is thought to be defective when its structure contains one or more exon deletions due to a genetic mutation. This disease primarily affects boys. Symptom onset is usually in early childhood, between ages 3 and 5. Muscle weakness can begin as early as age 3, first affecting the muscles of the hips, pelvic area, thighs and shoulders, and later the skeletal (voluntary) muscles in the arms, legs, and trunk. By the early teens, the heart and respiratory muscles also get affected, often requiring the use of assistive devices. Tests used to diagnose DMD vary from a blood test (measuring creatine kinase) to the muscle biopsy (measuring dystrophin protein levels), to the genetic testing (looking for the defective dystrophin gene). Standard of therapy is aimed at slowing the loss of muscle strength to maximize the quality of life, and involves physical therapy and medications, such as steroids: prednisone and deflazacort. Assistive devices for breathing difficulties may also be used later in the stages of disease progression. Exon skipping treatments are a novel approach used in the management of this disease. Note: The Introduction section is for your general knowledge and is not to be taken as policy coverage criteria. The rest of the policy uses specific words and concepts familiar to medical professionals. It is intended for providers. A provider can be a person, such as a doctor, nurse, psychologist, or dentist. A provider also can be a place where medical care is given, like a hospital, clinic, or lab. This policy informs them about when a service may be covered. Policy Coverage Criteria We will review specific intravenous (IV) and injectable drugs for medical necessity for all ages. For those age 13 and older, we also will review the site of service for medical necessity. Site of service is defined as the location where the drug is administered, such as a hospital-based outpatient setting, an infusion center, a physician’s office, or at home. Click here to be directed to the site of service review criteria. The following drugs addressed in this policy are subject to site of service review: • Exondys 51® (eteplirsen) • Vyondys 53® (golodirsen) Site of Service Medical Necessity Administration Medically necessary sites IV infusion therapy of various medical or biologic agents will of service be covered in the most appropriate, safe and cost-effective • Physician’s office site: • Infusion center • These are the preferred medically necessary sites of service for • Home infusion specified drugs. Hospital-based outpatient IV infusion therapy of various medical or biologic agents will setting be covered in the most appropriate, safe and cost-effective • Outpatient hospital IV site. infusion department Page | 2 of 22 ∞ Site of Service Medical Necessity Administration • Hospital-based outpatient This site is considered medically necessary for the first 90 days clinical level of care for the following: • The initial course of infusion of a pharmacologic or biologic agent OR • Re-initiation of an agent after 6 months or longer following discontinuation of therapy* Note: *This does not include when standard dosing between infusions is 6 months or longer This site is considered medically necessary when there is no outpatient infusion center within 50 miles of the patient’s home and there is no contracted home infusion agency that will travel to their home, or a hospital is the only place that offers infusions of this drug. This site is considered medically necessary only when the patient has a clinical condition which puts him or her at increased risk of complications for infusions, including any ONE of the following: • Known cardiac condition (eg, symptomatic cardiac arrhythmia) or pulmonary condition (eg, significant respiratory disease, serious obstructive airway disease, %FVC ≤ 40%) that may increase the risk of an adverse reaction • Unstable renal function which decreases the ability to respond to fluids • Difficult or unstable vascular access • Acute mental status changes or cognitive conditions that impact the safety of infusion therapy • A known history of severe adverse drug reactions and/or anaphylaxis to prior treatment with a related or similar drug Hospital-based outpatient These sites are considered not medically necessary for infusion setting and injectable therapy services of various medical and biologic Page | 3 of 22 ∞ Site of Service Medical Necessity Administration • Outpatient hospital IV agents when the site-of-service criteria in this policy are not infusion department met. • Hospital-based outpatient clinical level of care Drug Medical Necessity Antisense Oligonucleotides, IV Amondys 45® Amondys 45® (casimersen) may be considered medically (casimersen) IV necessary for male patients up to 21 years of age when: • Patient has the diagnosis of Duchenne Muscular Dystrophy AND • Patient has a confirmed mutation of the DMD gene that is amenable to exon 45 skipping: o Genetic testing is required to determine the specific DMD gene mutation for a definitive diagnosis AND • Patient is able to ambulate (with or without assistance*) and complete a 6-minute-walk distance test of at least 250 meters o Record of the baseline 6MWT is necessary for the initial review AND • Patient has been established on a stable dose of corticosteroids for at least 3 months AND • Dose is limited to 30 mg/kg IV once weekly Note: *Assistance not to include use of a wheelchair Exondys 51® (eteplirsen) Exondys 51® (eteplirsen) is subject to review for site of service IV administration. Exondys 51® (eteplirsen) may be considered medically necessary for male patients up to 19 years of age when: • Patient has the diagnosis of Duchenne Muscular Dystrophy AND Page | 4 of 22 ∞ Drug Medical Necessity Antisense Oligonucleotides, IV • Patient has a confirmed mutation of the DMD gene that is amenable to exon 51 skipping: o Genetic testing is required to determine the specific DMD gene mutation for a definitive diagnosis AND • Patient is able to ambulate (with or without assistance*) and complete a 6-minute-walk distance test of at least 250 meters o Record of the baseline 6MWT is necessary for the initial review AND • Patient has been established on a stable dose of corticosteroids for at least 3 months AND • Dose is limited to 30 mg/kg IV once weekly Note: *Assistance not to include use of a wheelchair Viltepso™ (vitolarsen) IV Viltepso™ (vitolarsen) may be considered medically necessary for male patients up to 10 years of age when: • Patient has the diagnosis of Duchenne Muscular Dystrophy AND • Patient has a confirmed mutation of the DMD gene that is amenable to exon 53 skipping: o Genetic testing is required to determine the specific DMD gene mutation for a definitive diagnosis AND • Patient is able to ambulate (with or without assistance*) with a time to stand of < 10 seconds AND • Patient has been established on a stable dose of corticosteroids for at least 3 months AND • Dose is limited to 80 mg/kg IV once weekly Note: *Assistance not to include use of a wheelchair Page | 5 of 22 ∞ Drug Medical Necessity Antisense Oligonucleotides, IV Vyondys 53® (golodirsen) Vyondys 53® (golodirsen) is subject to review for site of IV service administration. Vyondys 53® (golodirsen) may be considered medically necessary for male patients up to 15 years of age when: • Patient has the diagnosis of Duchenne Muscular Dystrophy AND • Patient has a confirmed mutation of the DMD gene that is amenable to exon 53 skipping: o Genetic testing is required to determine the specific DMD gene mutation for a definitive diagnosis AND • Patient is able to ambulate (with or without assistance*) and complete a 6-minute-walk distance test of at least 250 meters o Record of the baseline 6MWT is necessary for the initial review AND • Patient has been established on a stable dose of corticosteroids for at least 3 months AND • Dose is limited to 30 mg/kg IV once weekly Note: *Assistance not to include use of a wheelchair Corticosteroids, Oral Emflaza® (deflazacort) Emflaza® (deflazacort) may be considered medically necessary oral for the following labeled indication: • Treatment of Duchenne Muscular Dystrophy (DMD) in patients 2 years of age and older who have had an adequate trial* and treatment failure due to the lack of response or increase in adverse events with prednisone Note: *Adequate trial is defined as 3 continuous months of therapy. Page | 6 of 22 ∞ Drug Investigational As listed All other uses of the medications listed in this policy are considered investigational. Approval Criteria Initial authorization Amondys 45® (casimersen), Exondys 51® (eteplirsen), Viltepso™ (vitolarsen), and Vyondys 53® (golodirsen) can
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