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Forward The new introduction to gene therapy science has brought the healthcare world to a seminal crossroad as public and private payers endeavor to create new decision-making models for assessing the economic value and correct pricing of these exciting medical interventions. The impact of reimbursement decisions made by payers over the next few years regarding gene therapies could powerfully transform the lives of countless individuals afflicted with chronic, handicapping, or life-threatening disease. Gene therapy offers the potential to drastically alter the course of a disease or potentially provide a cure – not just continuing to treat the symptoms of the disease, which in the end, may never otherwise be cured. While the development of gene therapies is expanding at an almost exponential rate, eventual reimbursement is far from assured for those agents that ultimately receive regulatory approval. Monitoring the market access challenges faced by recently approved Zolgensma® for spinal muscular atrophy highlights payer and manufacturer conflicts in defining the value of therapy for specific populations. For Sarepta’s GMaX team, our market access strategies to date, and moving forward, are all calculated to proactively meet the anticipated decision-making needs of payers as they navigate the largely uncharted waters of gene therapy reimbursement. From a market access perspective, bringing innovative, one-time, high-cost therapies to the bedside requires certain critical goals be attained by Sarepta. Specifically, GMaX needs to help payers find a path to positive value assessment and reimbursement. This includes guiding with evidence the discussion regarding value in order to ensure Sarepta successfully achieves reimbursement – and at its target price. GMaX is working to determine the optimal pathways by identifying which reimbursement roads are not worth traveling and where new roads need to be built. 3 CONFIDENTIAL - FOR INTERNAL USE ONLY: DO NOT DISTRIBUTE