FDA grants Breakthrough Therapy Designation status for acalabrutinib

AstraZeneca and Acerta Pharma – its haematology R&D centre of excellence – have announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for acalabrutinib. This potential drug aims to treat patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. Acalabrutinib is a highly-selective, potent Bruton tyrosine kinase (BTK) inhibitor in development for the treatment of multiple B-cell cancers.

Acalabrutinib shows promise

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. They must also have demonstrated substantial improvement on a clinically-significant endpoint over currently available medicines and when there is significant unmet medical need.

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

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

Flavia Borellini, PhD, Acerta Pharma Chief Executive Officer, said: "This is an exciting regulatory milestone for our work in haematology. Acalabrutinib is a potent, irreversible BTK inhibitor with a high degree of specificity for its target.

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 the area of haematology. The acalabrutinib development programme includes both monotherapy and combination therapies for a broad range of blood cancers and solid tumours.