RP-A501 (AAV9.LAMP2B)

No price, economic model, cost-effectiveness analysis, budget impact analysis, or health technology assessment of RP-A501 has been published, and no payer coverage policy or prior authorization criteria naming RP-A501 was found. RP-A501 is investigational. The company presentation of October 2026 schedules completion of dosing in the 12-participant pivotal cohort for mid-2027, topline results for mid-2028, and a biologics license application for late 2028, and it marks launch in mid-2029. The Form 10-K filed in February 2026 states that the company has not commenced efforts to have its product candidates reimbursed. The company's population funnel starts from a modeled 10,000 to 11,000 people with Danon disease in the United States, about 4,000 of them male, and more than 20,000 across the United States and five European countries; the Form 10-K states a prevalence of 15,000 to 30,000 in the United States and Europe, and the page of the sponsored genetic testing program states approximately 15,000 in the United States, citing company data on file. The presentation reports more than 900 identified United States patients as of July 2026, a count that includes people who have received a heart transplant or have died, and a diagnosis rate under 10%. The company states that its initial focus is males with symptomatic hypertrophic cardiomyopathy and that estimated prevalence and identified patient records “do not establish the number of patients eligible for treatment”. It gives an illustrative, non-risk-adjusted global peak annual opportunity of more than $1 billion with the initial indication and more than $2 billion with potential lifecycle expansions, based on internal assumptions that include pricing, access, and uptake. One third-party forecast was identified: GlobalData analysts, cited in August 2025 by a publication GlobalData owns, estimated $364 million in sales by 2031 if the product is approved. The pivotal trial enrolls males aged 8 years and older with a pathogenic or likely pathogenic LAMP2 variant, left ventricular hypertrophy with a left ventricular ejection fraction of at least 50%, New York Heart Association class II to III, and elevated high-sensitivity troponin I, and it excludes an anti-AAV9 neutralizing antibody titer above 1:40 and prior organ transplantation. No source reports how many of the modeled or identified males meet these criteria. Confirming eligibility requires genetic testing and anti-AAV9 antibody testing: the 2026 Florida Medicaid independent laboratory fee schedule lists $392.06 for procedure code 81439, a hereditary cardiomyopathy sequencing panel, no published cost of anti-AAV9 antibody testing was found, and the prescribing information for Fayuvi, an approved AAV9 gene therapy, states that an FDA-authorized test for anti-AAV9 total binding antibodies is not currently available.

List prices of approved one-time gene therapies are the available price benchmarks. Among systemic adeno-associated virus products, the reported prices are $2.125 million for Zolgensma (wholesale acquisition cost, Novartis, 2019), $3.5 million for Hemgenix (2022 news report), $3.2 million for Elevidys (Sarepta Therapeutics, 2023), $2.7 million for Genglycos (wholesale acquisition cost reported by Managed Healthcare Executive, 2026), and $3.95 million for Fayuvi (list price reported by EMJ, 2026). Prices of other one-time gene therapies are $425,000 per eye for Luxturna (Spark Therapeutics, 2018), $2.59 million for Itvisma (reported by Managed Healthcare Executive, 2025), $3.0 million for Skysona (bluebird bio, 2022), $2.2 million for Casgevy (Vertex Pharmaceuticals, 2023), $3.1 million for Lyfgenia (bluebird bio, 2023), and $4.25 million for Lenmeldy (Orchard Therapeutics, 2024). A 2026 conference analysis of gene therapies authorized by the FDA as of January 1, 2026 found a wholesale acquisition cost at market entry for 26 therapies and a median inflation-adjusted cost of $2,316,863 for single-use therapies. The Institute for Clinical and Economic Review (ICER) stated 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 a 50/50 shared savings analysis. The cost offsets a model would consider are documented in other populations. For heart transplantation in the United States for all diagnoses, Milliman estimated 2025 billed charges of $1,918,700 per transplant and notes that billed charges are unlikely to equal the amount paid. Among pediatric heart transplant recipients for all diagnoses, total hospitalization costs in 2016 dollars were $767,221 in the most recent era studied, and $755,345 with a ventricular assist device compared with $457,086 without mechanical circulatory support. Adjusted total hospital charges for a defibrillator placement admission were $132,455 in adults without congenital heart disease, median charges per pediatric heart failure-related hospitalization were $72,087 in 2009, the mean cost of an adult hospitalization with primary heart failure was $11,552 in 2014, and annual median total medical costs for heart failure care were estimated at $24,383 per patient. In the International Danon Disease Registry, 17 of 52 males (32.7%) received a heart transplant and 17 (32.7%) a defibrillator over a median follow-up of 5.86 years. The 2023 international consensus statement expects extracardiac symptoms to be largely unaffected by gene therapy and lists the inability to re-treat among its challenges.

Payer management precedents quoted for other one-time gene therapies are pay-over-time and outcomes-based agreements of up to 5 years for Zolgensma, reimbursement of up to 80% of the cost of Zynteglo if transfusion independence is not achieved and maintained up to two years after infusion, outcomes-based contracts for Lyfgenia tied to hospitalizations with three years of follow-up, and delivery through networks of Qualified Treatment Centers for Zynteglo and Lenmeldy; Rocket Pharmaceuticals describes a limited network of Qualified Treatment Centers for its own approved gene therapy, Kresladi. The Cell and Gene Therapy Access Model of the Centers for Medicare & Medicaid Services is a voluntary model in which the agency negotiates outcomes-based agreements with manufacturers on behalf of state Medicaid agencies; its initial focus is gene therapy for sickle cell disease, and states began participation between January 2025 and January 2026. In August 2021, 18 large commercial health plans and state Medicaid programs imposed restrictions in 53.5% and 68.3%, respectively, of their coverage policies for 11 cell and gene therapies. Between 2017 and 2022, yearly Medicaid spending on gene and RNA therapies increased from $148.3 million to $879.7 million and yearly treatments from 327 to 1638, and starting in 2021, 11 states carved spinal muscular atrophy drugs out of managed Medicaid. Budget impact for RP-A501 therefore cannot be estimated from public data as of October 2026.