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

Generic, brand name and therapeutic class of product

11 evidence topics · 12 sources

Manufacturer: Rocket Pharmaceuticals

“Rocket Pharmaceuticals Inc.” (opens the source at this quote in a new tab)

“The Company has global commercialization and development rights to all of these product candidates under internally developed intellectual property rights and royalty-bearing license agreements.” (opens the source at this quote in a new tab)

“Drug product for the Phase 2 study is being produced in-house in our GMP manufacturing facility in Cranbury, NJ.” (opens the source at this quote in a new tab)

“Sponsor: Rocket Pharmaceuticals, Inc.” (opens the source at this quote in a new tab)

European applicant: Rocket Pharmaceuticals B.V.

“This decision is addressed to Rocket Pharmaceuticals, B.V., Kingsfordweg 151, 1043 GR – Amsterdam, The Netherlands.” (opens the source at this quote in a new tab)

Development code and descriptive name in the trial registry

“Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease” (opens the source at this quote in a new tab)

“RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene” (opens the source at this quote in a new tab)

“A Clinical Study Evaluating a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With DD” (opens the source at this quote in a new tab)

Name in the phase 1 publication

“Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease.” (opens the source at this quote in a new tab)

“RP-A501, a recombinant adeno-associated virus serotype 9 containing the transgene LAMP2B, which encodes an isoform of LAMP2” (opens the source at this quote in a new tab)

Active substance wording used by EMA

“adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene (RP-A501)” (opens the source at this quote in a new tab)

“adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene” (opens the source at this quote in a new tab)

Generic name in the FDA orphan drug designation record

“Adeno-associated virus serotype 9 vector containing the human lysosome-associated membrane glycoprotein 2 isoform B transgene (AAV9.LAMP2B)” (opens the source at this quote in a new tab)

Name under which EMA lists the product in the PRIME scheme

“RP-A501” (opens the source at this quote in a new tab)

“Name of the active substance, INN, common name, chemical name or company code.” (opens the source at this quote in a new tab)

Invented name in the European Union

“Invented name and authorisation status:” (opens the source at this quote in a new tab)

“The product is not authorised anywhere in the European Community.” (opens the source at this quote in a new tab)

LAMPLIGHT study branding
RecordQuoted identifier
Sponsor protocol number, phase 2 trial“RP-A501-0123”
Other identifier, phase 2 trial“LAMPLIGHT-2”
RecordQuoted identifier
Sponsor protocol number, phase 1 trial“RP-A501-0219”
Other identifier, phase 1 trial“LAMPLIGHT-1”

“LAMPLIGHT Danon Disease Gene Therapy Study” (opens the source at this quote in a new tab)

“The LAMPLIGHT Studies have study sites in the USA and internationally.” (opens the source at this quote in a new tab)

International nonproprietary name, United States adopted name, and brand name

No evidence found.

Therapeutic class: AAV9-based gene therapy

“RP-A501 is our investigational gene therapy for the treatment of DD and consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a full-length, wild-type version of the human LAMP2B transgene which is administered as a single intravenous (IV) infusion.” (opens the source at this quote in a new tab)

“RP-A501 is our investigational genetic medicine for the treatment of DD” (opens the source at this quote in a new tab)

“RP-A501 is the Company’s first adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon disease that is in development under a collaboration with the University of California San Diego School of Medicine.” (opens the source at this quote in a new tab)

Intervention type in the trial registry

“Genetic: RP-A501” (opens the source at this quote in a new tab)

“Biological: RP-A501” (opens the source at this quote in a new tab)