Exondys 51

Indicated For

Duchenne muscular dystrophy

About This Medication

# SareptAssist Patient Guide: How to Get Exondys 51 at Low or No Cost Exondys 51 (eteplirsen) is an FDA-approved treatment for Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the dystrophin gene amenable to exon 51 skipping, offered through Sarepta Therapeutics' **SareptAssist** patient support program.[1][2][5] This guide explains eligibility, application steps, and support options to help families access this therapy affordably. ## About Exondys 51 (eteplirsen) **Exondys 51** is an antisense oligonucleotide infused weekly (30 mg/kg dose) to treat DMD by skipping exon 51 in the dystrophin gene, increasing dystrophin production in skeletal muscle.[1][2][4] Approved under the FDA's accelerated approval pathway, it addresses an unmet need for certain DMD patients, though ongoing studies verify long-term clinical benefits like improved muscle function.[1][3] It's prescribed by neurologists experienced in DMD and requires genetic confirmation of eligibility.[4][9][10] DMD is a progressive genetic disorder causing muscle weakness, often leading to loss of ambulation. Exondys 51 aims to slow progression in eligible patients, typically children who are ambulatory (able to walk without assistive devices like wheelchairs).[4][10] Always review the full Prescribing Information and consult your doctor before starting.[1][2] ## Who Qualifies for SareptAssist? SareptAssist provides comprehensive support, including case managers to navigate insurance, infusions, and financial aid.[1][2][5] Qualification focuses on medical and genetic criteria rather than strict income limits: - Confirmed DMD diagnosis with exon 51-amenable mutation via genetic testing.[1][7][9][10] - Prescribed by a DMD specialist (e.g., neurologist).[4][9][10] - Ambulatory status (e.g., North Star Ambulatory Assessment score >17 or time-to-rise <7 seconds for initial therapy).[4] - No concurrent exon-skipping therapies or recent gene therapy like Elevidys.[4][10] **Income-based assistance** is available through Sarepta Therapeutics' patient assistance programs for uninsured or underinsured patients, though exact FPL thresholds are not publicly detailed—contact SareptAssist for personalized eligibility.[7] Commercial insurance patients may access copay help.[7] ## Income Eligibility Breakdown Specific income thresholds for SareptAssist are not explicitly listed online, as they are handled case-by-case via case managers.[1][5][7] General US patient assistance often follows **400-500% of the Federal Poverty Level (FPL)**, but confirm directly. Here's a sample table based on 2026 FPL guidelines (adjust for household size; annual figures): | Household Size | 400% FPL | 500% FPL | Notes | |---------------|----------|----------|-------| | Individual | $60,240 | $75,300 | Contact SareptAssist for exact criteria[7] | | Couple | $81,760 | $102,200| Income verification required | | Family of 3 | $103,280| $129,100| May include free drug for qualified uninsured | | Family of 4 | $124,800| $156,000| Copay cards for commercial plans[7] | *Table illustrative; actual eligibility varies. SareptAssist tailors support to your situation.*[7] ## Insurance Requirements Most insurers cover Exondys 51 as a **Tier 4 specialty drug** with **prior authorization (PA)** required.[7] Key needs: - Genetic confirmation of exon 51 mutation.[7][9][10] - Baseline ambulation proof (e.g., NSAA score, no wheelchair).[4][10] - No dose exceeding 30 mg/kg weekly.[9][10] Medicare Part D covers it, with options like the Prescription Payment Plan for copays.[3] Medicaid varies by state (e.g., Louisiana requires PA).[6][9] SareptAssist case managers help with PA appeals and coverage verification.[1][2][5] If denied, explore external reviews (e.g., Virginia's 120-day process).[7] ## Step-by-Step Application Process 1. **Confirm Eligibility**: Get genetic testing for exon 51 mutation and DMD diagnosis from a specialist.[1][7][10] 2. **Enroll in SareptAssist**: Visit exondys51.com or call case managers (details via site).[1][2][5] 3. **Prescribe and PA**: Doctor submits PA with records (ambulation, no contraindications).[4][7] 4. **Submit Financial Docs**: Proof of income, insurance denial (if applicable), ID.[7] 5. **Case Manager Review**: They coordinate insurance, copay aid, or free drug.[1][5] 6. **Approval**: Expect 6-12 months initial authorization.[4][10] Applications are straightforward; case managers guide you.[1] ## Timeline and Delivery - **PA Processing**: Varies by insurer (days to weeks); case managers expedite.[1][7] - **Initial Approval**: Up to 6-12 months therapy.[4][10] - **Delivery**: Via specialty pharmacies (e.g., Accredo); weekly infusions at centers.[7] - **Refills**: Reauthorization every 12 months with proof of ambulation/response.[4][10] SareptAssist ensures timely delivery and ongoing monitoring.[5] ## Alternatives if Denied - **Appeal PA**: Submit additional records (e.g., NSAA scores).[4][7] - **Other Exon-Skippers**: Vyondys 53 (exon 53), Amondys 45 (exon 45), Viltepso (exon 53)—if mutation matches (not combinable).[4] - **Gene Therapy**: Elevidys for different mutations, but restart criteria apply.[4][10] - **State Aid**: Medicaid, charity programs.[6][9] - **Reapply**: Update docs and recontact SareptAssist.[1] No biosimilars exist.[ ] ## Disclaimer This guide is for informational purposes only and not medical/financial advice. Eligibility, coverage, and costs vary. Consult your doctor, insurer, and SareptAssist. Policies change; verify current details. Sarepta Therapeutics does not guarantee assistance.[1][2][7] (Word count: 942)

Program information last verified: March 30, 2026