FDA grants 'breakthrough therapy designation' to AstraZeneca for acalabrutinib

AstraZeneca and its haematology research and development "centre of excellence", Acerta Pharma, announced on Tuesday that the US Food and Drug Administration granted ‘breakthrough therapy designation’ for ‘acalabrutinib’, for the treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.

The FTSE 100 company described acalabrutinib as a “highly-selective, potent” Bruton tyrosine kinase (BTK) inhibitor in development for the treatment of multiple B-cell cancers.

It said the breakthrough therapy designation was 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 medicines 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 current therapy,” said AstraZeneca’s chief medical officer and executive vice president of global medicines development, Sean Bohen.

“Breakthrough therapy designation for acalabrutinib will help us bring this potential new medicine to appropriate patients as quickly as possible.”

The company said 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.

“This is an exciting regulatory milestone for our work in haematology,” said Acerta Pharma chief executive officer Flavia Borellini.

“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.”

It was 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 haematology.

The board said the acalabrutinib development programme included both monotherapy and combination therapies for a broad range of blood cancers and solid tumours.