Evicenter
P&T meetings

RP-A501 (AAV9.LAMP2B)

Danon disease

Regulatory submission
BLA planned for late 2028
Last updated
300 sources

Product information and disease descriptionProduct descriptionProduct information

Access and distribution

12 evidence topics · 11 sources

Expanded access: not offered

“At this time, Rocket is not able to make its investigational gene therapy products available on an expanded (or early) access basis anywhere in the world.” (opens the source at this quote in a new tab)

“Enrollment in a clinical trial ensures that necessary data is generated to inform the use of new treatment options.” (opens the source at this quote in a new tab)

“Rocket reserves the right to revise this policy at any time, in accordance with the 21st Century Cures Act.” (opens the source at this quote in a new tab)

Current access through the pivotal trial: six recruiting sites in four countries

“This study has 6 locations” (opens the source at this quote in a new tab)

LocationQuoted site name
La Jolla, California, United States“University of California, San Diego”
Boston, Massachusetts, United States“Boston Children's Hospital”
Philadelphia, Pennsylvania, United States“Children's Hospital of Philadelphia”
Munich, Germany“German Heart Center Munich”
Florence, Italy“Meyer Children's Hospital”
London, United Kingdom“Great Ormond Street Hospital & UCL Institute of Cardiovascular Science”

“Clinical sites ready to enroll and dose” (opens the source at this quote in a new tab)

“Patients identified to support completion of clinical study” (opens the source at this quote in a new tab)

Referral into the trial through the LAMPLIGHT study website

“If you qualify, you will be directed to a leading Danon disease specialist participating in the clinical study.” (opens the source at this quote in a new tab)

“Have not received a heart transplant” (opens the source at this quote in a new tab)

“Please note: this study is not available to those currently participating in any other investigational clinical studies or who have received any other gene therapy treatment” (opens the source at this quote in a new tab)

Commercial preparation stated for RP-A501 (October 2026)

“commercialization plans, including the development of sales and marketing capabilities and relationships with treatment centers and other third parties” (opens the source at this quote in a new tab)

Patient identification: activities the company lists on its path to launch (October 2026)

“Aspiration: every person with HCM or a Danon disease signature and every at-risk relative of a confirmed Danon patient is genetically evaluated so no Danon diagnosis is missed” (opens the source at this quote in a new tab)

“Drive cardiologists to test young HCM & urgency with Danon signature” (opens the source at this quote in a new tab)

“Expand diagnostic infrastructure & access” (opens the source at this quote in a new tab)

“Empower patients & caregivers to seek genetic evaluation” (opens the source at this quote in a new tab)

“Multiply diagnoses through the family cascade” (opens the source at this quote in a new tab)

Patient identification: identified U.S. patients and diagnosis rate (data as of July 2026)

“Over 900 Identified US Danon Disease Patients” (opens the source at this quote in a new tab)

“Representing a current diagnosis rate under 10%” (opens the source at this quote in a new tab)

“Data as of July 2026; over 80% of patients reported in ICD-10 code and genetic testing lab data have been identified in the last 5 years” (opens the source at this quote in a new tab)

“Estimated prevalence and identified patient records do not establish the number of patients eligible for treatment.” (opens the source at this quote in a new tab)

Sponsored genetic testing: Mission: Genome Danon Disease Program

“Sponsored, no-charge genetic testing” (opens the source at this quote in a new tab)

“Through the program, individuals suspected of having Danon Disease have access to genetic testing and counseling to help bring them one step closer to a diagnosis and the appropriate clinical management.” (opens the source at this quote in a new tab)

“No patients, healthcare professionals, or payers, including government payers, are billed for this program.” (opens the source at this quote in a new tab)

“This program is available to patients 40 years of age or younger, in the United States, with a high suspicion of an inherited cardiomyopathy.” (opens the source at this quote in a new tab)

“All blood relatives of patients tested through this sponsored testing program and found to have a pathogenic or likely pathogenic variant are eligible” (opens the source at this quote in a new tab)

Qualified Treatment Center model described for Kresladi, the company's approved gene therapy

“The strategy is centered on a limited network of specialized Qualified Treatment Centers and an appropriately scaled commercial infrastructure, supporting patient access while maintaining disciplined investment.” (opens the source at this quote in a new tab)

“Rocket anticipates commercial availability and the start of patient onboarding in the fourth quarter of 2026.” (opens the source at this quote in a new tab)

“The Company is currently advancing commercial launch activities, including Qualified Treatment Center onboarding, manufacturing readiness, and patient access initiatives.” (opens the source at this quote in a new tab)

“Commercial product revenue will depend on the timing of treatment center activation and patient treatment.” (opens the source at this quote in a new tab)

Number, location, and qualification criteria of treatment centers for RP-A501

No evidence found.

Manufacturing and supply for commercialization

“This facility supports clinical development of our pipeline of AAV gene therapy product candidates from discovery through pivotal trials, with space for potential future expansion and commercialization.” (opens the source at this quote in a new tab)

“We plan to supplement our own direct manufacturing capabilities with third-party manufacturers for our AAV programs.” (opens the source at this quote in a new tab)

“We also do not have any current contractual relationships for the manufacture of commercial supplies of our product candidates if they become registered.” (opens the source at this quote in a new tab)

“Regulatory progress and production capacity can support pivotal study and commercialization” (opens the source at this quote in a new tab)

Sales and marketing capability

“If, in the future, we are unable to establish sales and marketing capabilities or enter into agreements with third parties to sell and market any of our product candidates, we may not be successful in commercializing those product candidates if and when they are approved.” (opens the source at this quote in a new tab)

Regulatory filings outside the United States: intent stated in September 2024

“Given the prevalence of Danon disease across regions, the Company plans to pursue regulatory filings concomitantly in the U.S. and ex-U.S.” (opens the source at this quote in a new tab)

“We are advancing promising clinical programs designed to support regulatory approvals in the U.S. and Europe, with potential expansion into Asia and beyond.” (opens the source at this quote in a new tab)

European trial authorization and release of drug product for EU use

“In January 2024, we received CTIS approval to include clinical trial sites in certain EU Member States.” (opens the source at this quote in a new tab)

“Successful QP audit of Rocket manufacturing facility; drug product cleared for EU use” (opens the source at this quote in a new tab)

Timing of a marketing authorisation application in the European Union

No evidence found.