LAMP2

Orphan designation: Adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene Treatment of Danon disease, 16/08/2023 Positive

Retrieved on: 
Dimanche, février 4, 2024
COMP, IRIS, European Commission, Committee, LAMP2, Patient, Danon disease, EMA, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene
    - Intended use
    - Treatment of Danon disease
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2826
    - Date of designation
    - Sponsor
    Rocket Pharmaceuticals B.V.
    Kingsfordweg 151
    1043 GR Amsterdam
    Noord-Holland
    Netherlands
    E-mail: [email protected]
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Rocket Pharmaceuticals Reports Third Quarter 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Lundi, novembre 6, 2023
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Consensus, Patient, Research, Busulfan, American College, CGMP, Genetic testing, Clinical trial, AAV, PDUFA, Incidence, IMPD, LV, Danon disease, Malignancy, Medication, Mortality, CMC, Conference, CTA, Acquisition, PKP2, Disease, JACC, Journal, Consensus decision-making, Priority review, Male, PKD, Natural history, MHRA, Disorder, EMA, Arrhythmia, Journal of the American College of Cardiology, LAMP2, Safety, Investment, Diagnosis, BLA, Pediatric Blood & Cancer, Pharmaceutical industry, Medical device, Security (finance), Fine chemical, G&A

Supported peer-reviewed expert consensus paper to increase awareness of diagnosis and clinical management of patients with Danon Disease.

Key Points: 
  • Supported peer-reviewed expert consensus paper to increase awareness of diagnosis and clinical management of patients with Danon Disease.
  • Highlights of the publication include a review of diagnosing Danon Disease emphasizing the importance of genetic testing upon clinical suspicion, natural history, management recommendations and recent advances in potential gene therapy treatment.
  • Rocket is working towards initiation of Phase 2 pivotal trial activities in Europe and the UK.
  • As of September 30, 2023, Rocket had cash, cash equivalents and investments of $437.2 million.

Rocket Pharmaceuticals Reaches FDA Alignment on Pivotal Phase 2 Trial Design for RP-A501 in Danon Disease

Retrieved on: 
Mardi, septembre 12, 2023
Research, FDA, Genetics, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, NYHA, Male, CMS, LV, Troponin, IMPD, CMC, Center for Biologics Evaluation and Research, CTA, KCCQ, Disorder, Natriuresis, Medication, New York Heart Association Functional Classification, Food and Drug Administration Amendments Act of 2007, Clinical trial, CGMP, Patient, Heart transplantation, Immunohistochemistry, Mortality, Danon disease, Death, Food, Safety, Disease, Pharmaceutical industry, Medical device, EU, LAMP2

The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.

Key Points: 
  • The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.
  • A global natural history study will serve as an external comparator and run concurrently to the Phase 2 pivotal trial.
  • In-house manufacturing has been completed with sufficient high-quality drug product produced to fully supply the Phase 2 pivotal study.
  • Additionally, Rocket has secured an ICD-10 code from CMS for LAMP2 deficiency in Danon Disease

Rocket Pharmaceuticals Reports Second Quarter 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Jeudi, août 10, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, University of California, San Diego Performance-Based Skills Assessment, Communication, Advanced Therapy Medicinal Product, LV, University, PKP2, Marketing, European Medicines Agency, Survival, Clinical trial, Bone marrow, UCSD, Arrhythmia, AAV, Disorder, Medication, Fast track (FDA), EMA, ATMP, MMC, BAG3, Investigational New Drug, Invitae, Patient, IRB, Investment, CGMP, LAMP2, Genetic testing, Cardiomyopathy, FDA, Death, BLA, IND, Gene, Safety, Pharmaceutical industry, Vaccine, Fine chemical, Cryptocurrency, G&A, Danon disease

“The second quarter of 2023 marked a period of strong forward momentum across our cardiovascular AAV and hematology LV portfolios highlighted by the BLA submission for our LAD-I program, Rocket’s first product filing.

Key Points: 
  • “The second quarter of 2023 marked a period of strong forward momentum across our cardiovascular AAV and hematology LV portfolios highlighted by the BLA submission for our LAD-I program, Rocket’s first product filing.
  • ATMP designation is intended for medicines that offer groundbreaking new opportunities and allows for a single evaluation and authorization procedure.
  • Initiating two-patient pediatric safety run-in for RP-A501 pivotal study; approaching final alignment with FDA on primary composite endpoint to support accelerated approval.
  • As of June 30, 2023, Rocket had cash, cash equivalents and investments of $307.0 million.

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial and Operational Results

Retrieved on: 
Lundi, février 27, 2023
Research, Other Health, General Health, Pharmaceutical, Oncology, Biometrics, Genetics, Clinical Trials, Science, Cardiology, Biotechnology, Health, Other Science, CGMP, Fanconi anemia, Bone marrow, Cell, Acquisition, Lighting, Empire, Hemolysis, CD18, Regenerative Medicine Advanced Therapy, AAV, PKD, Novartis, BM, Danon disease, Senior, Biologics license application, Chief of Naval Staff (Pakistan), Patient, LV, Heart Failure Society of America, Rare disease, Pyruvate kinase deficiency, Transplant, Kaplan–Meier estimator, Schering-Plough, Center for International Blood and Marrow Transplant Research, Eli Lilly, Investment, Heart failure, Congress, Gene, Disease, MMC, Quality of life, Survival, LAMP2, PKP2, Disorder, DCM, BLA, FDA, ASH, Conference, FA, Medication, Society, Pharmaceutical industry, Medical device, Cryptocurrency, EU, G&A

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2022.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2022.
  • The Company previously presented positive data from the study at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022.
  • Based on the data from the Phase 2 pivotal Fanconi Anemia trial, Rocket anticipates submitting a BLA in the fourth quarter of 2023.
  • As of December 31, 2022, Rocket had cash, cash equivalents and investments of $399.7 million.

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-A501 Gene Therapy for Danon Disease

Retrieved on: 
Mardi, février 7, 2023
Health, FDA, Genetics, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, AAV, NYHA, Troponin, Danon disease, Natural history, Biologics license application, Disorder, Drug development, LAMP2, Breakthrough therapy, Patient, Clinical trial, News, Food, BNP, RP, New York Heart Association Functional Classification, Disease, 21st Century Cures Act, Orphan drug, Adolescence, Risk assessment, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, LV, Medication, Pharmaceutical industry, Medical device

“Today’s exciting RMAT designation demonstrates recognition from the FDA of the early meaningful benefit of RP-A501 in Danon Disease and its potential to deliver lifesaving treatment for patients,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.

Key Points: 
  • “Today’s exciting RMAT designation demonstrates recognition from the FDA of the early meaningful benefit of RP-A501 in Danon Disease and its potential to deliver lifesaving treatment for patients,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.
  • As previously disclosed, the Company anticipates pursuing a single arm, open-label trial with a biomarker-based composite endpoint and a natural history comparator.
  • Results from the Phase 1 program represent one of the most comprehensive investigational gene therapy datasets for any cardiac condition.
  • Overall, the results demonstrate improvements and/or normalization across multiple quantifiable parameters that cardiologists use in clinical practice to enable risk assessment and treatment decisions.