lovo-cel (gene therapy)
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Product Information
Scientific name: lovotibeglogene autotemcel (lovo-cel)
Brand name: Lyfgenia™
RESPONSIBLE: bluebird bio Inc. -
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 countryCompletion 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 countryCompletion date: February 2024
Scope of the Study / Aim: No VOC for between 6 – 18 consecutive months post-infusionHGB-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 countryCompletion date: April 2027
Scope of the Study / Aim: No VOC for between 6 – 18 consecutive months post-infusion13 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 countryCompletion 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
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)
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:
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- 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.
- 27 QTCs are ready to receive patient referrals.
- Information is available at mybluebirdsupport.com.
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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