Stage (next event)
Pre-BLA meeting with the FDA
Catalyst Info & Data Links
TITLE: Etranacogene dezaparvovec (AMT-061) for Hemophilia B - BLA Submission
ClinicalTrials.gov (NCT03569891): HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
WHAT IS THE CATALYST EVENT?
WHEN WILL THE EVENT (OR DID THE EVENT) OCCUR?
1. Q1 2021
11-2020: Corporate Update (see slides 7-14)
2019: Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B. Blood advances, 3(21), 3241-3247.
2014: Long-term safety and efficacy of factor IX gene therapy in hemophilia B. New England Journal of Medicine, 371(21), 1994-2004.
Mechanism of Action
MECHANISM OF ACTION
Etranacogene dezaparvovec, also known as AMT-061, consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). AAV5-based gene therapies have been demonstrated to be safe and well tolerated in many clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with the Company’s AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates. Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.
Disease prevalence: ~6,000 patients in the United States and ~14,000 patients in Europe
Updated by HC
Prior Data (click to view full image)
Trial Design / Revenue (click to view full image)
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