Spinraza (Nusinersen) Drug Prior Authorization Protocol (Medical Benefit & Part B Benefit)

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1 Line of Business: All Lines of Business Effective Date: August 16, 2017 Spinraza (Nusinersen) Drug Prior Authorization Protocol (Medical Benefit & Part B Benefit) This policy has been developed through review of medical literature, consideration of medical necessity, generally accepted medical practice standards, and approved by the IEHP Pharmacy and Therapeutics Subcommittee. Policy/Criteria: Eligibility Criteria: Check CCS Eligibility Diagnosis: a. Spinal muscular atrophy (SMA type 1, SMA type 2 or SMA type 3) with genetic testing result confirming the mutation of the SMN1 protein on the 5q chromosome (also known as classic SMA), as evidenced by one of the following: i. Homozygous gene deletion or mutation ii. Compound heterozygous mutation Specialist: Neurologist, or other Prescribers in consultation with a physician who specializes in treatment of SMA Criteria: a. Clinical review by IEHP pharmacist b. One of the following: i. Member does not yet have SMA-associated symptoms [i.e. pre-symptomatic] (e.g. failure to meet motor milestones such as head control, sitting, ability to kick, rolling, crawling, standing and walking, etc.) but has documentation of the presence of one to three copies of SMN2 protein ii. Documentation of SMA-associated symptoms (e.g. failure to meet motor milestones such as head control, sitting, ability to kick, rolling, crawling,

2 standing and walking, etc.) before 12 years of age (i.e. SMA type 1, type 2 or type 3) iii. Documentation of SMA-associated symptoms (e.g. failure to meet motor milestones such as head control, sitting, ability to kick, rolling, crawling, standing and walking, etc.) with impaired but functional SMN1 protein c. Documentation of one of the following: i. For non-sitters: CHOP Intend or Hammersmith Infant Neurological Exam- Part 2 (HINE-2) ii. For sitters: Hammersmith Functional Motor Scale (HFMS) iii. For walkers: the 6 minute walk test or the 10 meter run/walk test d. Documented baseline platelet count 50,000 cells per microliter e. Member does not have a co-existing terminal condition or a condition in which the risk of Spinraza treatment outweighs the potential benefit f. Spinraza can be safely administered intrathecally. For older members with SMA scoliosis, the drug may only be authorized if member has: i. Scoliosis without spine surgery ii. Post-spine surgery with preserved window of accessibility by intrathecal injection iii. Post-spine surgery without window of accessibility with surgical placement of an indwelling catheter or establishment of a new window for IT accessibility Care Coordination: a. Interdisciplinary comprehensive care, including physical therapy, respiratory care and nutritional support, is highly recommended. Please task to care management team for care coordination if deemed necessary Authorization Duration: a. 6 months Re-Authorization Criteria: a. Documentation of one of the following (completed prior to each re-authorization): i. For non-sitters: CHOP Intend or Hammersmith Infant Neurological Exam- Part 2 (HINE-2) ii. For sitters: Hammersmith Functional Motor Scale (HFMS) iii. For walkers: the 6 minute walk test or the 10 meter run/walk test

3 b. Documentation of meeting therapeutic goals including motor function improvement or stabilization of motor function loss compared to the predicted natural trajectory of disease c. Documented platelet count 50,000 cells per microliter within the past 2 months d. Requested dosage and administration are consistent with the FDA recommendation Formulary Status: Non-formulary, PA applies Clinical Justification: Background: SMA is a rare, neurodegenerative disease caused by a genetic defect, which leads to progressive loss of motor neuron function and severe muscular atrophy. Patients with severe SMA can become paralyzed requiring mechanical ventilation, tube feeding and nursing care. SMA type 1 (infantile SMA) is the most common and severe form of SMA (onset <6 months of age). Average time to death or ventilation dependence is 13.5 months. With SMA type 2 (onset 6-18 months of age), patients may reach motor milestone more slowly. Typically, patients can sit up but would lose this ability over time, and may never walk. With SMA type 3 (onset after 1 year of age), patients may gain the ability to walk but would progressively lose this ability to walk with time. Spinraza (nursinersen) is FDA approved for SMA due to SMN1 protein mutation on the 5q chromosome (aka classic SMA) resulting in inadequate production of SMN protein Consensus Statement for Standard of Care in Spinal Muscular Atrophy Recommended optimal, aggressive multidisciplinary care, including orthopedic/rehabilitation, pulmonary, gastrointestinal/nutrition care, along with psychological and social support. Therapy should be individualized based on patient s functional level. Clinical Trial Infantile-Onset SMA In a multicenter, randomized, double-blind, sham-procedure controlled study, symptomatic infants (n=121) at 7 months of age or younger with confirmed diagnosis of SMA were randomized to receive either Spinraza or sham injection. Baseline demographics, disease duration, SMN2 copy number and age at symptom onset were balanced between the two groups.

4 The proportion of subjects who were motor milestone responders was significantly higher in the Spinraza group than placebo, based on a pre-planned interim analysis. Phase 3 Clinical Trial Later-Onset SMA In a Phase 3, multicenter, randomized, double-blind, sham-procedure controlled study, 126 symptomatic, non-ambulatory children at 2-12 years of age with confirmed diagnosis of SMA who experienced symptom onset at greater than 6 months of age were randomized to receive either Spinraza or sham injection. The Spinraza group demonstrated a high statistically significant improvement in motor function from baseline to 15 months in the HFMSE score (+3.9 vs. -1.0, p= ). Primary endpoints results at the end of the study were consistent with results observed at the interim analysis. Favorable safety profile was noted, as the majority of the adverse events were considered to be either related to SMA disease, common events in the general population, or events related to the lumbar puncture procedure. No children discontinued the study due to adverse events. Adapted from the Spinraza FDA Medical Review

5 MOA Indication Pediatric Indication Usual Dosing Spinraza (nusinersen) Antisense oligonucleotide (ASO) that increases exon 7 inclusion in SMN2 mrna transcripts and production of full-length SMN protein Spinal muscular atrophy (SMA) Safety and efficacy established for newborns to 17 years old 12 mg (5 ml) per administration First three loading doses administered at 14-day intervals. Fourth loading dose administered 30 days after the third dose. Maintenance dose administered once every four months thereafter. Administration Intrathecal bolus injection over 1 to 3 minutes by, or under direction of, healthcare professionals experienced in performing lumbar punctures. Dosage adjustment in renal impairment None; quantitative spot urine protein testing recommended at baseline, prior to each dose, and as clinically needed PK/PD Distribution: CNS, skeletal muscle, liver, and kidney Metabolism: exonuclease (3 - and 5 )-mediated hydrolysis t1/2 = days in CSF, days in plasma Excretion: renal Contraindications None Drug-drug interaction None Common Side effects Lower and upper respiratory tract infection, and constipation Monitoring Platelet count, prothrombin time/activated partial thromboplastin time, and quantitative spot urine protein testing at baseline, prior to each dose, and as clinically indicated. Warnings Thrombocytopenia and coagulation abnormalities Renal toxicity Pregnancy No adequate data Lactation Storage and stability How supplied No data Protect from light and store refrigerated at 2-8 C. Do not freeze. Injection 12 mg/5 ml (2.4 mg/ml) single-dose vial free of preservatives

6 References: 1. Department of Health Care Services. All County California Children Services (CCS) Administrators, Medical Consultants, and State Systems of Care Division Staff. Nusinersen (Spinraza) March 9, Spinraza (nusinersen) injection for intrathecal use [package insert]. Cambridge, MA: Biogen, Inc; December Spinraza [Prescribing Information]. Biogen Inc., Cambridge, MA; Retrieved from df/spinraza-prescribing-information.pdf. Accessed July 12, Wang, CH, Finkel, RS, et al. Consensus statement for standard of care in spinal muscular atrophy. Journal of child neurology Aug; 22(8): Change Control Date Change 08/16/2017 Revised criteria Added re-authorization criteria