Clinical Trial Updates (SCD)

lovo-cel (gene therapy)

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  • Product Information

    Product Information

    Scientific name: lovotibeglogene autotemcel (lovo-cel)
    Brand name: N/A
    RESPONSIBLE: bluebird bio Inc.

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Last update: 22/6/2023

    Northstar (HGB-205)

    Trial Name: Northstar (HGB-205)
    Code: NCT02151526 
    Phase: 1B
    Eligible patient diagnosis: TDT or SCD (all genotypes) (adult & paediatric/adolescent ; ages 5 – 35)
    No. of patients enrolled: 7→ 4 [3 with βE/β0 genotype, 1 with IVS-I110 mutation] & 3 [with SCD] (Last update: Jan 2022)
    Study Sites: 1 Sites per country

    Completion date: February 2019
    Scope of the Study / Aim: No VOC or ACS post-infusion for at least 24 months
    Results & more information: Thompson A, et al. (2019)

    HGB-206 (Group C)

    Trial Name: HGB-206 (Group C)
    Code: NCT02140554 
    Phase: 1/2
    Eligible patient diagnosis: SCD (adult and adolescent) (ages 12 – 50)
    No. of Patients enrolled: 50 [anticipated] [45 infused – Last update: 20/12/2022] Recruitment completed (Last update: 23/09/2023)
    Study Sites: 11 Sites per country

    Completion date: February 2024
    Scope of the Study / Aim: No VOC for between 6 – 18 consecutive months post-infusion

    HGB-210

    Trial Name: HGB-210
    Code: NCT04293185
    Phase: 3
    Eligible patient diagnosis: SCD (adult and paediatric/adolescent) (ages 2 – 50
    No. of Patients enrolled: 35 [anticipated] [2 infused – Last update: 21/6/2023] (Last update: 24/3/2023)
    Study Sites: 9 Sites per country

    Completion date: April 2027
    Scope of the Study / Aim: No VOC for between 6 – 18 consecutive months post-infusion

    13 Years Follow-up Study

    Trial Name: Long-term follow-up study (LTF-307)
    Code: NCT04628585
    Phase: : Long-term follow-up study of Phase 1/2 studies [HGB-205, HGB-206], or Phase 3 studies [HGB-210
    Eligible patient diagnosis: SCD patients who participated in HGB-205, HGB206, HGB-210 (ages 2 – 53)
    No. of Patients enrolled: 85 [anticipated] (Last update: 24/5/2023)
    Study Sites: 14 Sites Sites per country

    Completion date: January 2038
    Scope of the Study / Aim: After completing the parent clinical study (approximately 2 years), patients will be followed for an additional 13 years for a total of 15 years post infusion. Monitoring for malignancies, immune-related adverse events, haematologic disorders and neurologic disorders.

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

     

    Additional notable points:

    • EMA: N/A
    • FDA: Orphan drug designation (2014), fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare paediatric disease designation, Priority Review for BLA submission accepted (Jun 2023) – PDUFA Target Date: 20 December 2023
    • MHRA: N/A

Update: 30 September 2023

  • The Institute for Clinical and Economic Review (ICER) determined that lovo-cel will be cost-effective if priced between $1.35M to $2.05M. ICER has made recommendations to encourage companies to set prices toward lower end of this range to facilitate access and affordability across all insurance systems (in the USA).
  • The FDA will not hold an advisory committee to discuss lovo-cel before making a decision (expected for 20 December 2023).

Sources: https://icer.org/news-insights/press-releases/icer-publishes-final-evidence-report-on-gene-therapies-for-sickle-cell-disease/
https://www.fiercebiotech.com/biotech/bluebirds-lovo-cel-flies-closer-approval-fda-rules-out-adcom-ahead-december-decision


Update: 30 June 2023

  •  BLA application submitted to FDA (24 April 2023) for lovo-cel for patients with SCD who are 12 years and older and have a history of VOCs. The submission is based on the results of 36 patients in HGB-206 Group C clinical trial (median 32 months follow-up) and 2 patients in the HGB-210 study (18 months followup).
  • Commercial launch is anticipated in early 2024 if approved.
  • FDA has accepted Priority Review for BLA submission and set a PDUFA (the date by which the FDA must respond to the application) target date of 20 December 2023.

Sources: bluebird bio Reports First Quarter 2023 Financial Results and Highlights Operational Progress – bluebird bio, Inc.
https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-biosubmits-biologics-license-application-bla-fda-0
https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bioannounces-fda-priority-review-biologics-license-1

 

Update: 31 March 2023

  • Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease, with more than 50 patients treated and multiple patients followed for more than six years.
  • On track to submit biologics license application (BLA) to the U.S. Food and Drug Administration in Q1 2023.
  • If approved by FDA, anticipated commercial launch in early 2024.

Sources: https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bioprovides-update-commercial-launch-progress-program
https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bioreports-fourth-quarter-and-full-year-2022-financial 

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