Product information and disease description Product description Product information

American hospital formulary service (AHFS), or other drug classification

6 evidence topics · 10 sources

AHFS Pharmacologic-Therapeutic Classification, Anatomical Therapeutic Chemical code, and USP category assigned to RP-A501

No evidence found.

EU classification: gene therapy medicinal product

Field	Quoted record
Public description of active substance	“Recombinant Adeno-associated virus serotype 9 vector containing the human-lysosome-associated membrane glycoprotein 2 isoform B transgene (AAV9.LAMP2B)”
Indication	“Treatment of Danon Disease”
Classification conclusion	“the EMA/CAT considers that the Product falls within the definition of a gene therapy medicinal product.”
Outcome of classification	“GTMP”

EMA PRIME list: substance type and therapeutic area

Field	Quoted record
Substance type	“Advanced Therapy Medicinal Product”
Therapeutic area	“Cardiovascular Diseases”

EMA paediatric investigation plan: therapeutic area

“Congenital, familial and genetic disorders” (opens the source at this quote in a new tab)

FDA regenerative medicine advanced therapy designation: eligibility definition quoted by the company

“A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition.” (opens the source at this quote in a new tab)
“RP-A501 is the first cardiac gene therapy to receive RMAT designation from the FDA” (opens the source at this quote in a new tab)

Regulatory product class in the United States: biologic reviewed under a Biologics License Application

“FDA approval of a Biologics License Application for RP-A501 for the treatment of Danon Disease” (opens the source at this quote in a new tab)

ICD-10-CM code for the disease: E74.05, which the company states it secured in 2023

“Additionally, Rocket has secured an ICD-10 code from CMS for LAMP2 deficiency in Danon Disease.” (opens the source at this quote in a new tab)

“In 2023, Rocket secured an ICD-10 code from the Centers for Medicare and Medicaid Services (CMS) to document patients with LAMP2 deficiency in Danon disease.” (opens the source at this quote in a new tab)

“In July 2023, US Dept. Health and Human Services approved IDC 10 Code for DD (E74.05)” (opens the source at this quote in a new tab)

“ICD-10 code for Danon available from Q 4 2023” (opens the source at this quote in a new tab)

Index entry	Quoted record
Deficiency term	“lysosome-associated membrane protein 2 [LAMP2]”
Code	“E74.05”