US FDA backs AstraZeneca drug for pancreatic cancer
Cambridge pharmaceutical giant AstraZeneca and Merck & Co have won a US FDA award of Orphan Drug Designation for Lynparza (olaparib) for the treatment of pancreatic cancer.
The core IP behind the technology arose from discoveries made by Professor Steve Jackson’s Cancer Research UK-funded team in Cambridge. Lynparza is bolt-on the next blockbuster drug with Cambridge roots. This is the fourth Orphan Drug Designation in the US for AstraZeneca and Merck’s Lynparza.
Pancreatic cancer is a rare, life-threatening disease that accounts for some three per cent of all cancers in the US.
Due to the late onset of symptoms patients are often diagnosed after the cancer has progressed to locally advanced or metastatic stages of the disease. Five-year survival rates remain low – 8.5 per cent in the US, for example.
Sean Bohen, AstraZeneca’s executive VP for Global Medicines Development and chief medical officer said: “Pancreatic cancer is an area of significant unmet medical need. This is especially true for patients with metastatic disease where the benefits of current treatment options are very limited.”
Roy Baynes, chief medical officer, at MSD Research Laboratories, said the FDA grant of Orphan Drug Designation was a positive step for patients with pancreatic cancer and continued to reinforce the importance of the collaboration in bringing Lynparza to more patients in need.
ODD status was granted for the treatment of ovarian cancer in October 2013. Earlier this year an amended ODD status was granted to include both fallopian tube and primary peritoneal cancers.
The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.
The use of Lynparza in pancreatic cancer is being assessed in the ongoing Phase III POLO trial, which is testing Lynparza as maintenance monotherapy vs placebo in patients with germline BRCA-mutated metastatic pancreatic cancer whose disease has not progressed following 1st-line platinum-based chemotherapy. Results from the POLO trial are expected in the first half of 2019.
Lynparza was the first in class PARP inhibitor and the first targeted treatment to potentially exploit DNA damage response (DDR) pathway deficiencies, such as BRCA mutations, to preferentially kill cancer cells.
Specifically, in vitro studies have shown that Lynparza-induced cytotoxicity may involve inhibition of PARP-enzymatic activity and increased formation of PARP-DNA complexes, resulting in DNA damage and cancer cell death.
Lynparza, which has the broadest clinical development programme of any PARP inhibitor, is being investigated in a range of DDR-deficient tumour types, and is the foundation of AstraZeneca's industry-leading portfolio of compounds targeting DDR mechanisms in cancer cells.