Blood Cells.

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for acalabrutinib for the treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.

Mantle cell lymphoma (MCL) is an aggressive B-cell non-Hodgkin lymphoma (NHL) with poor prognosis.[1][2][3][4] MCL accounts for approximately 3% to 6% of new NHL cases in Western countries each year, with an annual incidence of 0.5 per 100,000 persons and an estimated prevalence of 3.5/100,000.[2][5] The median age at diagnosis is 68 years, with a 3:1 male predominance.[2]

Acalabrutinib is an investigational, highly selective, potent, covalent inhibitor of Bruton tyrosine kinase (BTK) being developed by AstraZeneca and Acerta Pharma (a member of the AstraZeneca Group), for the treatment of multiple B-cell cancers. In pre-clinical trials, researchers noted minimal off-target activity observed.[6][7][8]

The acalabrutinib development program includes both monotherapy and combination therapy strategies in chronic lymphocytic leukemia (CLL), MCL, Waldenstr?m macroglobulinemia, follicular lymphoma, diffuse large B-cell lymphoma, and multiple myeloma, as well as monotherapy and combination trials in solid tumors.

In total, more than 25 acalabrutinib clinical trials with more than 2,000 patients are underway or have completed. Acalabrutinib was granted Orphan Drug Designation by the FDA for the treatment of patients with MCL in September 2015.

Breakthrough Therapy
The Breakthrough Therapy Designation is designed to expedite the development and regulatory review of new medicines that are intended to treat a serious condition and that have shown encouraging early clinical results, which demonstrate substantial improvement on a clinically-significant endpoint over available therapies and when there is significant unmet medical need.

?New treatments are urgently needed for people with mantle cell lymphoma who relapse or do not respond to therapy. Breakthrough Therapy Designation for acalabrutinib will help us bring this potential medicine to appropriate patients as quickly as possible,? noted Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca.

Clinical data
The FDA granted Breakthrough Therapy Designation based on the totality of clinical data from the acalabrutinib development program, including data from the Phase II ACE-LY-004 clinical trial in patients with relapsed or refractory MCL.

?This is an exciting regulatory milestone for our work in hematology. 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,? explained Flavia Borellini, PhD, Acerta Pharma Chief Executive Officer.

This is the fifth Breakthrough Therapy Designation that AstraZeneca has received from the FDA for an oncology medicine since 2014, and the first for the Company in hematology. The acalabrutinib development program includes both monotherapy and combination therapies in a broad range of blood cancers and solid tumors.


Last editorial review: August 1, 2017

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