On August 1, 2017 AstraZeneca and its haematology research and development centre of excellence, Acerta Pharma, reported that the US 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 (Press release, AstraZeneca, AUG 1, 2017, View Source [SID1234519952]). Acalabrutinib is a highly-selective, potent Bruton tyrosine kinase (BTK) inhibitor in development for the treatment of multiple B-cell cancers. Schedule your 30 min Free 1stOncology Demo! 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 medicines and when there is significant unmet medical need.
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
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. If approved, it could be a clinically-meaningful treatment option for patients with this devastating disease."
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 haematology. The acalabrutinib development programme includes both monotherapy and combination therapies for a broad range of blood cancers and solid tumours.
About mantle cell lymphoma (MCL)
Mantle cell lymphoma (MCL) is an aggressive B-cell non-Hodgkin lymphoma (NHL) with poor prognosis.[i],[ii],[iii],[iv] 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,[v] The median age at diagnosis is 68 years, with a 3:1 male predominance.2
About acalabrutinib
Acalabrutinib is a highly selective, potent, covalent inhibitor of Bruton tyrosine kinase (BTK) with minimal off-target activity observed in pre-clinical trials.[vi],[vii],[viii] This potential new medicine is in development for the treatment of multiple B-cell and other cancers. The acalabrutinib development programme includes both monotherapy and combination therapy strategies in chronic lymphocytic leukaemia (CLL), MCL, Waldenström macroglobulinemia (WM), follicular lymphoma, diffuse large B-cell lymphoma, and multiple myeloma, as well as monotherapy and combination trials in solid tumours. 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 and by the European Commission in March 2016 for the treatment of patients with CLL, MCL and WM. Acalabrutinib is a potential new medicine not approved for any current use.