US FDA grants breakthrough therapy designation for AstraZeneca’s MCL treatment


AstraZeneca’s acalabrutinib has received breakthrough therapy designation from the US Food and Drug Administration (FDA) for the treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.

MCL is an aggressive B-cell non-Hodgkin lymphoma (NHL) with poor prognosis.

Acalabrutinib is an investigational, highly selective, potent Bruton tyrosine kinase (BTK) inhibitor developed by the company’s haematology research and development centre of excellence, Acerta Pharma.

AstraZeneca chief medical officer and Global Medicines Development executive vice-president Sean Bohen 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 potential new therapy is currently under development for the treatment of multiple B-cell cancers and other cancers.

The FDA’s breakthrough therapy designation is based entirely on the clinical data obtained from the acalabrutinib development programme, including data from the Phase II ACE-LY-004 clinical study that involved patients with relapsed or refractory MCL.

"This is an exciting regulatory milestone for our work in haematology."

Acerta Pharma chief executive officer Dr Flavia Borellini 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. If approved, it could be a clinically meaningful treatment option for patients with this devastating disease.”

AstraZeneca’s acalabrutinib development programme involves both monotherapy and combination therapies for a wide range of blood cancers and solid tumours.

In September 2015, the FDA granted orphan drug designation for the medicine to treat MCL.


Image: Mantle cell lymphoma. Photo: courtesy of Gabriel Caponetti.