This designation is expected to expedite the development and regulatory review of acalabrutinib.
Acalabrutinib, also known as ACP-196, is an oral therapy that targets the enzyme Bruton tyrosine kinase, or BTK, which plays a key role in the survival and proliferation of malignant B-cells. It was developed by Acerta Pharma, a member of the AstraZeneca group.
Preclinical-trial studies have demonstrated that acalabrutinib is a potent inhibitor of BTK, and has little impact other than on the enzyme.
“New treatments are urgently needed for people with mantle cell lymphoma who relapse or do not respond to current therapy,” Sean Bohen, the chief medical officer of AstraZeneca, said in a press release. “Breakthrough Therapy Designation for acalabrutinib will help us bring this potential new medicine to appropriate patients as quickly as possible.”
Results of a number of clinical trials supported the FDA designation. They included interim findings from the ongoing Phase 2 ACE-LY-004 trial (NCT02213926).
Acerta is conducting the trial in the United States and United Kingdom. The main objective is to evaluate the safety and effectiveness of acalabrutinib as a stand-alone treatment against relapsed or refractory mantle cell lymphoma. Researchers hope to enroll 120 adults in the study.
The company is testing acalabrutinib as both a stand-alone therapy and in combination with standard-of-care treatments for of multiple B-cell cancers. They include chronic lymphocytic leukemia, diffuse large B-cell lymphoma, and follicular lymphoma. Acerta has also tested it in several solid cancers.
More than 25 clinical trials are involved in the development program, with more than 2,000 patients enrolled.
“This is an exciting regulatory milestone for our work in hematology,” said Dr. Flavia Borellini, chief executive officer of Acerta Pharma. “Acalabrutinib is a potent, irreversible BTK inhibitor with a high degree of specificity for its target. If approved, it could be a clinically meaningful treatment option for patients with this devastating disease.”
The FDA gave acalabrutinib orphan drug status as a mantle cell lymphoma treatment in 2015. The European Commission granted it orphan drug status in March 2016 for treating patients with mantle cell lymphoma, chronic lymphocytic leukemia, and Waldenström macroglobulinemia.