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A chimeric antigen receptor (CAR) T-cell therapy, bb2121, has received breakthrough therapy designation from the US Food and Drug Administration (FDA) and was granted access to the European Medicines Agency’s (EMA’s) PRIority MEdicines (PRIME) program.
The CAR T-cell therapy is designed to target B-cell maturation antigen in previously treated patients with multiple myeloma.
bb2121 is being developed by bluebird bio, Inc., and Celgene Corporation.
The EMA’s and FDA’s decisions on bb2121 were based on preliminary data from an ongoing phase 1 study, CRB-401 (NCT02658929).
Results from this study were presented at the 2017 ASCO Annual Meeting (abstract 3010).
The study enrolled patients with relapsed and/or refractory multiple myeloma. As of the May 4, 2017 data cut-off, 21 patients had been enrolled.
Patients had a median of 7 prior lines of therapy (range, 3-14). Their previous treatments included lenalidomide and bortezomib (100%), pomalidomide and carfilzomib (91%), daratumumab (71%), and autologous stem cell transplant (100% at least once).
Twenty-nine percent of patients were refractory to bortezomib, lenalidomide, carfilzomib, pomalidomide, and daratumumab.
Patients received a conditioning regimen of cyclophosphamide and fludarabine, followed by an infusion of bb2121 at 1 of 4 doses: 50 x 106, 150 x 106, 450 x 106 and 800 x 106 CAR+ T cells.
All 21 patients were evaluable for safety.
The most common treatment-emergent grade 3-4 adverse events were cytopenias commonly associated with the lymphodepletion regimen, as well as grade 3 events of hyponatremia (n=4), upper respiratory infection (n=2), syncope (n=2), and cytokine release syndrome (CRS, n=2).
In all, 71% of patients (15/21) had CRS, mostly grade 1 and 2. For the 2 patients with grade 3 CRS, it resolved within 24 hours. To manage CRS, 4 patients received tocilizumab, and 1 (with grade 2) received steroids as well.
Eighteen patients were evaluable for efficacy, and the overall response rate was 89% (16/18).
There were 4 complete responses—2 in the 150 x 106 cohort, 1 in the 450 x 106 cohort, and 1 in the 800 x 106 cohort. The complete responder in the 450 x 106 cohort ultimately died of cardiopulmonary arrest that was deemed unrelated to treatment.
Five patients had a partial response—2 in the 450 x 106 cohort and 1 in each of the other cohorts. Seven patients had a very good partial response—5 in the 450 x 106 cohort and 1 each in the 150 x 106 cohort and 800 x 106 cohort.
Updated data from this study are scheduled to be presented at the 2017 ASH Annual Meeting (abstract 740).
About breakthrough designation
The FDA’s breakthrough designation is intended to expedite the development and review of new treatments for serious or life-threatening conditions.
The designation entitles the company developing a therapy to more intensive FDA guidance on an efficient and accelerated development program, as well as eligibility for other actions to expedite FDA review, such as rolling submission and priority review.
To earn breakthrough designation, a treatment must show encouraging early clinical results demonstrating substantial improvement over available therapies with regard to a clinically significant endpoint, or it must fulfill an unmet need.
About PRIME
The EMA launched its PRIME program to enhance support for the development of medicines that target an unmet medical need.
The program involves enhanced interaction and early dialogue with developers of promising medicines to optimize development plans and speed up evaluation so these medicines can reach patients earlier.
PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. To be accepted for PRIME, a medicine must have demonstrated the potential to benefit patients with unmet medical needs based on early clinical data. 
A chimeric antigen receptor (CAR) T-cell therapy, bb2121, has received breakthrough therapy designation from the US Food and Drug Administration (FDA) and was granted access to the European Medicines Agency’s (EMA’s) PRIority MEdicines (PRIME) program.
The CAR T-cell therapy is designed to target B-cell maturation antigen in previously treated patients with multiple myeloma.
bb2121 is being developed by bluebird bio, Inc., and Celgene Corporation.
The EMA’s and FDA’s decisions on bb2121 were based on preliminary data from an ongoing phase 1 study, CRB-401 (NCT02658929).
Results from this study were presented at the 2017 ASCO Annual Meeting (abstract 3010).
The study enrolled patients with relapsed and/or refractory multiple myeloma. As of the May 4, 2017 data cut-off, 21 patients had been enrolled.
Patients had a median of 7 prior lines of therapy (range, 3-14). Their previous treatments included lenalidomide and bortezomib (100%), pomalidomide and carfilzomib (91%), daratumumab (71%), and autologous stem cell transplant (100% at least once).
Twenty-nine percent of patients were refractory to bortezomib, lenalidomide, carfilzomib, pomalidomide, and daratumumab.
Patients received a conditioning regimen of cyclophosphamide and fludarabine, followed by an infusion of bb2121 at 1 of 4 doses: 50 x 106, 150 x 106, 450 x 106 and 800 x 106 CAR+ T cells.
All 21 patients were evaluable for safety.
The most common treatment-emergent grade 3-4 adverse events were cytopenias commonly associated with the lymphodepletion regimen, as well as grade 3 events of hyponatremia (n=4), upper respiratory infection (n=2), syncope (n=2), and cytokine release syndrome (CRS, n=2).
In all, 71% of patients (15/21) had CRS, mostly grade 1 and 2. For the 2 patients with grade 3 CRS, it resolved within 24 hours. To manage CRS, 4 patients received tocilizumab, and 1 (with grade 2) received steroids as well.
Eighteen patients were evaluable for efficacy, and the overall response rate was 89% (16/18).
There were 4 complete responses—2 in the 150 x 106 cohort, 1 in the 450 x 106 cohort, and 1 in the 800 x 106 cohort. The complete responder in the 450 x 106 cohort ultimately died of cardiopulmonary arrest that was deemed unrelated to treatment.
Five patients had a partial response—2 in the 450 x 106 cohort and 1 in each of the other cohorts. Seven patients had a very good partial response—5 in the 450 x 106 cohort and 1 each in the 150 x 106 cohort and 800 x 106 cohort.
Updated data from this study are scheduled to be presented at the 2017 ASH Annual Meeting (abstract 740).
About breakthrough designation
The FDA’s breakthrough designation is intended to expedite the development and review of new treatments for serious or life-threatening conditions.
The designation entitles the company developing a therapy to more intensive FDA guidance on an efficient and accelerated development program, as well as eligibility for other actions to expedite FDA review, such as rolling submission and priority review.
To earn breakthrough designation, a treatment must show encouraging early clinical results demonstrating substantial improvement over available therapies with regard to a clinically significant endpoint, or it must fulfill an unmet need.
About PRIME
The EMA launched its PRIME program to enhance support for the development of medicines that target an unmet medical need.
The program involves enhanced interaction and early dialogue with developers of promising medicines to optimize development plans and speed up evaluation so these medicines can reach patients earlier.
PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. To be accepted for PRIME, a medicine must have demonstrated the potential to benefit patients with unmet medical needs based on early clinical data.
A chimeric antigen receptor (CAR) T-cell therapy, bb2121, has received breakthrough therapy designation from the US Food and Drug Administration (FDA) and was granted access to the European Medicines Agency’s (EMA’s) PRIority MEdicines (PRIME) program.
The CAR T-cell therapy is designed to target B-cell maturation antigen in previously treated patients with multiple myeloma.
bb2121 is being developed by bluebird bio, Inc., and Celgene Corporation.
The EMA’s and FDA’s decisions on bb2121 were based on preliminary data from an ongoing phase 1 study, CRB-401 (NCT02658929).
Results from this study were presented at the 2017 ASCO Annual Meeting (abstract 3010).
The study enrolled patients with relapsed and/or refractory multiple myeloma. As of the May 4, 2017 data cut-off, 21 patients had been enrolled.
Patients had a median of 7 prior lines of therapy (range, 3-14). Their previous treatments included lenalidomide and bortezomib (100%), pomalidomide and carfilzomib (91%), daratumumab (71%), and autologous stem cell transplant (100% at least once).
Twenty-nine percent of patients were refractory to bortezomib, lenalidomide, carfilzomib, pomalidomide, and daratumumab.
Patients received a conditioning regimen of cyclophosphamide and fludarabine, followed by an infusion of bb2121 at 1 of 4 doses: 50 x 106, 150 x 106, 450 x 106 and 800 x 106 CAR+ T cells.
All 21 patients were evaluable for safety.
The most common treatment-emergent grade 3-4 adverse events were cytopenias commonly associated with the lymphodepletion regimen, as well as grade 3 events of hyponatremia (n=4), upper respiratory infection (n=2), syncope (n=2), and cytokine release syndrome (CRS, n=2).
In all, 71% of patients (15/21) had CRS, mostly grade 1 and 2. For the 2 patients with grade 3 CRS, it resolved within 24 hours. To manage CRS, 4 patients received tocilizumab, and 1 (with grade 2) received steroids as well.
Eighteen patients were evaluable for efficacy, and the overall response rate was 89% (16/18).
There were 4 complete responses—2 in the 150 x 106 cohort, 1 in the 450 x 106 cohort, and 1 in the 800 x 106 cohort. The complete responder in the 450 x 106 cohort ultimately died of cardiopulmonary arrest that was deemed unrelated to treatment.
Five patients had a partial response—2 in the 450 x 106 cohort and 1 in each of the other cohorts. Seven patients had a very good partial response—5 in the 450 x 106 cohort and 1 each in the 150 x 106 cohort and 800 x 106 cohort.
Updated data from this study are scheduled to be presented at the 2017 ASH Annual Meeting (abstract 740).
About breakthrough designation
The FDA’s breakthrough designation is intended to expedite the development and review of new treatments for serious or life-threatening conditions.
The designation entitles the company developing a therapy to more intensive FDA guidance on an efficient and accelerated development program, as well as eligibility for other actions to expedite FDA review, such as rolling submission and priority review.
To earn breakthrough designation, a treatment must show encouraging early clinical results demonstrating substantial improvement over available therapies with regard to a clinically significant endpoint, or it must fulfill an unmet need.
About PRIME
The EMA launched its PRIME program to enhance support for the development of medicines that target an unmet medical need.
The program involves enhanced interaction and early dialogue with developers of promising medicines to optimize development plans and speed up evaluation so these medicines can reach patients earlier.
PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. To be accepted for PRIME, a medicine must have demonstrated the potential to benefit patients with unmet medical needs based on early clinical data.