The FDA granted Olaparib (Lynparza) an orphan drug designation by for the treatment of patients with pancreatic cancer,
Pancreatic cancer is a rare, life-threatening disease that accounts for about 3% 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 in the US at 8.5%.
Sean Bohen, Executive Vice President, 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, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, at MSD Research Laboratories, 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.”
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 following the expanded US approval of Lynparza in August 2017 for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer, who are in a complete or partial response to platinum-based chemotherapy. 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 ongoing, randomized, multicenter, double-blind, placebo-controlled phase III POLO trial (NCT02184195) is currently evaluating olaparib as maintenance therapy in patients with germline BRCA1/2-mutated metastatic pancreatic cancer whose disease has not progressed following first-line platinum-based chemotherapy.
A total 145 patients have been randomized 3:2 to receive oral olaparib tablets at 300 mg twice daily as maintenance therapy versus placebo also twice daily. Randomization occurs within 6 weeks following last chemotherapy dose and olaparib/placebo treatment will begin within 4 to 8 weeks of the last chemotherapy dose. Following randomization, patients have weekly clinical visits for the first 4 weeks of treatment, then every 4 weeks while on study treatment.
Treatment will continue until objective radiological disease progression. Following progression, patients will be followed for second progression every 8 weeks, and then for survival until final analysis.
Eligible patients were previously treated for metastatic disease and have not progressed following completion of at least 16 weeks of frontline platinum-based chemotherapy. Additionally, patients must have a known deleterious or suspected deleterious germline BRCA mutation. Those who were previously treated with a PARP inhibitor were excluded.
The primary endpoint is progression-free survival. Secondary endpoints are overall survival, time from randomization to second progression or death, objective response rate, disease control rate, safety, and tolerability. Results from this study are expected in the first half of 2019.
