AstraZeneca and Merck have secured the orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for their poly ADP-ribose polymerase (PARP) inhibitor Lynparza (olaparib) to treat pancreatic cancer.
Lynparza has been designed to exploit DNA damage response (DDR) pathway deficiencies, like the BRCA mutations, to preferentially eliminate cancer cells.
Especially, in vitro studies have demonstrated that Lynparza-induced cytotoxicity could involve inhibition of PARP-enzymatic activity and increased formation of PARP-DNA complexes, leading to DNA damage and cancer cell death.
Pancreatic cancer, for which Lynparza has been given the FDA the orphan drug designation, is considered to be a rare, life-threatening condition, pancreatic cancer makes up around 3% of all cancers in the US, according to the American Cancer Society.
The FDA’s orphan drug designation is given to drugs that are being developed for the treatment, diagnosis or prevention of rare diseases or disorders that affect less than 200,000 people in the US.
AstraZeneca global medicines development executive vice president and chief medical officer Sean Bohen 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.”
Lynparza, which has been co-developed by the two pharma majors, is being evaluated for its use in pancreatic cancer in an ongoing phase 3 trial called POLO.
The late-stage POLO trial is assessing the PARP inhibitor as maintenance monotherapy in comparison to placebo in patients having germline BRCA-mutated metastatic pancreatic cancer whose condition has not progressed after first-line platinum-based chemotherapy.
The data from the POLO trial is anticipated to come out in the first half of 2019.
MSD Research Laboratories senior vice president and global clinical development head Roy Baynes said: “Pancreatic cancer is a relatively less common, but life-threatening, form of cancer. The FDA granting Orphan Drug Designation is a positive step for patients with pancreatic cancer and continues to reinforce the importance of our collaboration in bringing Lynparza to more patients in need.”
In May 2018, Lynparza was approved by the European Medicines Agency (EMA) for the treatment of patients with platinum-sensitive relapsed ovarian cancer.
The approval was given for 300mg twice daily of Lynparza tablets as a maintenance therapy for patients with platinum-sensitive relapsed high-grade, epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in complete response or partial response to platinum-based chemotherapy, irrespective of BRCA status.