Product information and disease description Place of product in therapy Approaches to treatment

Other product development or post-marketing obligations required by the FDA

4 evidence topics · 5 sources

EMA paediatric investigation plan: agreed clinical studies

Measure	Quoted description
Study 1	“Open-label, non-randomised, single dose trial to evaluate safety and efficacy of RP-A501 in male patients from 8 years to less than 18 years of age (and adults) with Danon disease with any confirmed lysosome-associated membrane protein 2 (LAMP2) mutation(s) and cardiac involvement.”
Study 2	“Open-label, single-arm, single dose trial to evaluate safety and efficacy of RP-A501 in male patients from 8 years to less than 18 years of age (and adults) with Danon disease”“with a pathogenic or likely pathogenic LAMP2 mutation(s) and evidence of left ventricular hypertrophy with preserved systolic function.”
Study 3	“Open-label, single-arm, single dose trial to evaluate safety and efficacy of RP-A501 in male patients from 3 to less than 8 years of age with Danon disease with a pathogenic or likely pathogenic LAMP2 mutation(s) and evidence of left ventricular hypertrophy with preserved systolic function.”
Study 4	“Open-label, single-arm, single dose trial to evaluate safety and efficacy of RP-A501 in female patients with Danon disease with a pathogenic or likely pathogenic LAMP2 mutation(s) and evidence of left ventricular hypertrophy with preserved systolic function.”
Study 5	“Observational study with both retrospective and prospective data collection designed to describe the natural history of Danon disease including the signs and symptoms, key clinical events, and impact of the disease on quality of life.”
Extrapolation plan	“Studies 1, 2 and 5 are part of an extrapolation plan covering the paediatric population from 3 years to less than 8 years of age, as agreed by the PDCO.”

EMA paediatric investigation plan: deferral granted and completion by December 2034

“Deferral for one or more measures contained in the paediatric investigation plan: Yes” (opens the source at this quote in a new tab)
“Date of completion of the paediatric investigation plan: By December 2034” (opens the source at this quote in a new tab)
“A deferral for adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene (RP-A501), solution for infusion, intravenous use, the details of which are set out in the opinion of the Paediatric Committee of the European Medicines Agency annexed hereto, together with its appendices, is hereby granted.” (opens the source at this quote in a new tab)

EMA paediatric investigation plan: compliance check status

Field	Quoted record
Compliance check done	“No”

Evidence after accelerated approval: company statements on full approval and confirmatory trials

“Additional secondary endpoints will include natriuretic peptides, Kansas City Cardiomyopathy Questionnaire (KCCQ), New York Heart Association (NYHA) class, event free survival to 24 months and treatment emergent safety events.” (opens the source at this quote in a new tab)
“These endpoints could support a full approval with longer-term follow-up.” (opens the source at this quote in a new tab)

“As a condition of accelerated approval, the FDA may impose specific obligations with defined timelines, including to perform confirmatory clinical trials with due diligence.” (opens the source at this quote in a new tab)
“Even if we do receive accelerated approval or conditional approval, we may not ultimately receive full approval from the regulatory agencies.” (opens the source at this quote in a new tab)

“any requirements for additional studies or post-approval confirmatory trials” (opens the source at this quote in a new tab)

Post-marketing requirements or commitments issued by the FDA for RP-A501

Not applicable.