Clinical Trial Updates (SCD)

lovo-cel (gene therapy)

  • Product Information

    Product Information

    Scientific name: lovotibeglogene autotemcel (lovo-cel)
    Brand name: Lyfgenia™
    RESPONSIBLE: bluebird bio Inc.

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    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: 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 ; Approved for the treatment of sickle cell disease (SCD) in patients 12 years and older who have a history of VOCs (Dec 2023)
    • MHRA: N/A

Update: 30 September 2024

  • 4 patient cell collections have been completed (14 August 2024)
  • Qualified Treatment Centre (QTC) network: 70 centres activated to –date (adult & paediatric) for thalassaemia (14 August 2024)

Source: https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-reports-second-quarter-2024-results-and-highlights

 

Update: 30 June 2024

  • 1 patient cell collections have been completed (9 May 2024)
  • Qualified Treatment Centre (QTC) network: 64 centres activated to –date (adult & paediatric) for thalassaemia (9 May 2024)
  • Pricing & Reimbursement: Multiple outcomes-based agreements in place ; discussions ongoing with Medicaid agencies

 

 

 

 

 

 

 

Sources: bluebird bio Reports First Quarter 2024 Results and Highlights Operational Progress and 2024 Guidance – bluebird bio, Inc.
bluebird bio Announces Completion of First Cell Collection for LYFGENIA™ Gene Therapy – bluebird bio, Inc.

 

Update: 31 March 2024

  • 1st patient start is imminent (26 March 2024)
  • Qualified Treatment Centre (QTC) network: 49/62 centres activated for ZYNTEGLO/thalassaemia to –date (adult & paediatric) are receiving SCD patients referrals for LYFGENIA (26 March 2024)
  • Pricing & Reimbursement: The company has signed its first Medicaid outcomes-based agreement with the state of Michigan, and with an additional 4 commercial payers. Approximately 50 percent of individuals living with sickle cell disease in the U.S. are insured by Medicaid.
  • HGB-210 (evaluating lovo-cel for patients under 12 years old): enrolment anticipated to be completed in Q4 2024.

Sources: https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-reports-fourth-quarter-and-2023-annual-results-and
https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-provides-update-commercial-launch-progress-0
https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-first-outcomes-based-agreement-medicaid

 

Update: 20 December 2023

  • FDA: Approved for the treatment of sickle cell disease (SCD) in patients 12 years of age and older who have a history of VOCs (Dec 2023)
  • The approval is based on data from patients from the Phase 1/2 HGB-206 study.
  • Safety data from 54 patients who initiated stem cell collection.

Efficacy data from 36 patients in the Phase ½ HGB-206 Group C study following enhancements to the treatment and manufacturing processes made through the course of the clinical development program. 32 patients were evaluated, including 8 adolescent patients, showing that severe VOCs were resolved in 30/32 patients (94%) and 28/32 patients (88.2%) experienced no VOCs at all.

The company plans to make the therapy available in early 2024 in the USA.

Pricing & Reimbursement:

    • The wholesale acquisition cost of LYFGENIA in the U.S. has been announced as set at $3.1M
    • The company is in advanced discussions with the nation’s largest commercial payers and more than 15 Medicaid agencies, representing 80% of individuals with sickle cell disease in the S.

Delivery:

LYFGENIA will be available through an established network of Qualified Treatment Centers (QTCs), which receives specialized training to administer complex gene therapies like LYFGENIA.

New data

Data presented at the 65th ASH Annual Congress (9–12 December 2023) in San Diego (USA) showed that:

  • 34/47 patients treated in HGB-206 Group C and HGB-210 were evaluable (data cut-off: February 13, 2023) and had a maximum of 60-month follow-up. Of these:
    – 32/34 patients (94%) experienced complete resolution of sVOEs, maintained for a median of 35.8 months.
    – 30/34 patients (88.2%) experienced complete resolution of all VOEs, maintained for a median of 35.8 months.

Patients who experienced VOEs at any time post-treatment through long-term follow-up (n=8) experienced a significant reduction in VOE frequency and severity compared to before treatment.

  • All 8 patients experienced a reduction in VOEs of at least 50%.
  • Hospital days and admissions were reduced by as much as 100% (annualized median hospital days reduced from 15.75 to 2.20).
  • Results of a sub-analysis of data from adolescent patients, presented for the first time, showed complete resolution of VOEs and sVOEs in 10/10 (100%) of patients during the 6–18 month enrollment period.

Sources: https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-fda-approval-lyfgeniatm-lovotibeglogene
https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-details-plans-commercial-launch-lyfgeniatm-gene
https://investor.bluebirdbio.com/news-releases/news-release-details/long-term-follow-data-bluebirds-gene-therapy-program-sickle-cell
https://ash.confex.com/ash/2023/webprogram/Paper174229.html

 

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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