RP-A501 (AAV9.LAMP2B)

No economic model, cost-effectiveness analysis, budget impact analysis, or health technology assessment of RP-A501 was identified, and Rocket Pharmaceuticals has announced no price. The company presentation of October 2026 schedules a biologics license application for late 2028 and marks a launch in mid-2029. The same presentation estimates 10,000 to 11,000 people with Danon disease in the United States (about 4,000 male and 6,000 to 7,000 female) and more than 20,000 across the United States and five European countries, reports more than 900 identified United States patients (a diagnosis rate under 10%), and gives an illustrative, non-risk-adjusted global peak annual opportunity of more than $1 billion for males with hypertrophic cardiomyopathy and more than $2 billion with expansion to other segments. The Form 10-K filed in February 2026 states a prevalence of 15,000 to 30,000 in the United States and Europe and states that the company has not begun efforts to obtain reimbursement. One third-party forecast, attributed to GlobalData in August 2025, is $364 million in sales by 2031. The company owes a low single digit royalty on net sales to the University of California San Diego and royalties in the high single digits to low teens to REGENXBIO, with 20% of any priority review voucher fees; Rocket sold a voucher from another product for gross proceeds of $180 million in June 2026.

Wholesale acquisition costs or list prices reported at launch for 11 one-time gene therapies range from $425,000 per eye (Luxturna, 2018) to $4.25 million (Lenmeldy, 2024). The systemic AAV products among them are Zolgensma at $2.125 million (2019), Hemgenix at $3.5 million (2022), Elevidys at $3.2 million (2023), Genglycos at $2.7 million (2026), and Fayuvi, an AAV9 product approved in September 2026, at $3.95 million. An analysis of 27 gene therapies authorized by January 1, 2026 found a wholesale acquisition cost for 26 and a median inflation-adjusted price of $2,316,863 for the 25 single-use entries. The Institute for Clinical and Economic Review set a value-based price benchmark of $1.1 million to $1.9 million for Zolgensma and a health benefit price benchmark of approximately $3,000,000 for Hemgenix after capping cost offsets at $150,000 per year, and its methods for single and short-term therapies add optimistic and conservative benefit scenarios and two shared-savings analyses. Milliman estimates 2025 billed charges of $1,918,700 for a heart transplant in the United States, the procedure the company describes as the only definitive treatment.

Payer management precedents are outcomes-based and pay-over-time contracts (five years for Zolgensma; a refund of up to 80% within two years for Zynteglo; three years for Lyfgenia), qualified treatment center networks (Lenmeldy; Kresladi, the first approved product of Rocket Pharmaceuticals), and the Cell and Gene Therapy Access Model of the Centers for Medicare & Medicaid Services, which began with sickle cell disease gene therapies and state participation from January 2025. In August 2021, 53.5% of commercial and 68.3% of Medicaid coverage policies for 11 cell and gene therapies imposed restrictions. Medicaid spending on gene and RNA therapies for rare inherited conditions rose from $148.3 million in 2017 to $879.7 million in 2022, and 11 states carved spinal muscular atrophy drugs out of managed Medicaid beginning in 2021.