New treatment for infants with spinal muscular atrophy

On May 24, 2019, the U.S. Food and Drug Administration approved Zolgensma (onasemnogene abeparvovec), a new gene replacement therapy. Zolgensma is approved to treat children less than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, a leading genetic cause of infant mortality.¹ Sufferers of this rare genetic neuromuscular flaw experience abnormal spinal muscle function and develop significant motor deficiencies before six months of age. The goal of Zolgensma is to correct the defect and restore normal muscle development and function.

AveXis/Novartis, the manufacturer of Zolgensma, determines the facilities in the United States authorized to administer this gene therapy. Customers should verify if a facility participates in their plan’s Cigna network. If a customer receives treatment out-of-network, depending on their plan design, coverage may be excluded or subject to the plan’s out-of-network cost-share requirements.

Approximately 450 to 500 infants are born with SMA in the U.S. annually.² Depending upon their specific genetic and clinical characteristics, a subset of these infants will be candidates for treatment with Zolgensma.

The wholesale acquisition cost of Zolgensma is $2,125,000.³ AveXis is working with payers to offer pay-over-time options up to five years.⁴

Zolgensma must be administered by a health care provider and will be eligible for coverage as a medical benefit (not a pharmacy benefit) under Cigna-administered health plans.⁵

Learn more

1.  Zolgensma Prescribing Information. May 2019. <https://www.avexis.com/content/pdf/prescribing_information.pdf.>
2. Verhaart IEC, Robertson A, et al. J Neurol. 2017 Jul;264(7):1465-1473.
3. Zolgensma Product Fact Sheet. AveXis, Inc. May 2019.
4. AveXis Announces Innovative Zolgensma® Gene Therapy Access Programs for US Payers and Families. AveXis, Inc. Press Release. May 24, 2019. <http://investors.avexis.com/phoenix.zhtml?c=254285&p=irol-newsArticle&ID=2399690.>
5. Medical plans vary, but in general to be eligible for coverage a drug must be approved by the Food and Drug Administration (FDA), prescribed by a health care professional, purchased from a licensed pharmacy and medically necessary. Coverage is subject to plan copayment, coinsurance or deductible requirements. Refer to your plan documents for costs and complete details of coverage.