Breakthrough Therapy Designation Granted to Orelabrutinib by FDA for Relapsed/Refractory MCL


The FDA granted orelabrutinib breakthrough therapy designation for the treatment of relapsed or refractory mantle cell lymphoma.

Orelabrutinib has been granted breakthrough therapy designation (BTD) by the FDA for the treatment of relapsed or refractory mantle cell lymphoma (R/R MCL), according to a press release from the company developing the Bruton tyrosine kinase (BTK) inhibitor, InnoCare Pharm.1

Orelabrutinib is highly selective for both B-cell malignancies as well as autoimmune diseases. This therapy is currently approved by the China National Medical Products Administration (NMPA) for 2 indications: relapsed or refractory chronic lymphocytic leukemia and R/R MCL.

“We are very proud that orelabrutinib was granted BTD after obtaining Orphan Drug Designation. We will continue to uphold the concept of ‘Science drives innovation for the benefit of patients’ and accelerate clinical trials for multiple indications of orelabrutinib in China and the rest of the world to benefit patients worldwide,” Jasmine Cui, PhD, co-founder, chairwoman, and CEO of InnoCare, said in a press release.

One phase 2 study presented at the 2020 American Society of Hematology (ASH) Annual Meeting looked on orelabrutinib monotherapy in 106 Chinese patients with R/R MCL .2 With a median follow-up time of 16.4 months, 87.9% reached the primary end point of objective response. The secondary end points of duration of response (DOR) and progression-free survival were not reached. Frequently reported adverse effects were thrombocytopenia, neutropenia, leukopenia, and hypertension.

Additionally, the NMPA has approved a clinical trial of orelabrutinib with rituximab (Rituxan), cyclophosphamide, doxorubicin, vincristine, and presnisone (R-CHOP) for previously untreated patients with MCL versus placebo plus R-CHOP. The primary end point is PFS with secondary end points or objective response rate, DOR, and overall survival.3

“Data from trials with orelabrutinib have shown sustained efficacy in the treatment of R/R MCL patients,” Jun Zhu, MD, PhD, professor and director of the Department of Internal Medicine and Lymphoma at the Peking University Cancer Hospital in China, said in a press release.4 “We believe the improved safety profile resulting from high selectivity and the convenience of daily oral administration will help make orelabrutinib a favorable treatment option for patients of B-cell malignancies.”


1. Innocare Announces Breakthrough Therapy Designation of Orelabrutinib by US FDA for Treatment of R/R MCL. News Release. InnoCare. June 28, 2021. Accessed June 29, 2021.

2. Innocare Presents Latest Clinical Data of Orelabrutinib at the 62nd Annual Meeting of ASH. News Release. InnoCare Pharma. December 7, 2020. Accessed June 29, 2021.

3. InnoCare Announces Approval of Orelabrutinib in Combination with R-CHOP as First-Line Therapy for Mantle Cell Lymphoma in China. News Release. InnoCare Pharma. February 5, 2021. Accessed June 29, 2021.

4. InnoCare Announces the Approval of Orelabrutinib in China for Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma and Relapsed/Refractory Mantle Cell Lymphoma. News Release. InnoCare Pharma. December 28, 2020. Accessed June 29, 2021.

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