AstraZeneca announced Lynparza (olaparib) has been granted orphan drug designation (ODD) in Japan for the maintenance treatment of germline BRCA-mutated (gBRCAm) curatively unresectable pancreatic cancer. Lynparza is co-developed and co-commercialized with MSD.
The Japanese Ministry of Health, Labour and Welfare grants ODD to medicines intended for the treatment of diseases that affect fewer than 50,000 patients in Japan and for which there is a high unmet medical need.
Pancreatic cancer has the lowest survival rate of the most common cancers and is the only major cancer with a five-year survival rate below 10% in almost every country.
"Japan has the fifth-highest incidence of pancreatic cancer worldwide and patients have seen limited treatment advances over the last few decades. This designation is an important step forward in bringing the first targeted medicine to biomarker-selected patients with advanced pancreatic cancer in Japan," José Baselga, Executive Vice President, R&D Oncology, said.
"A pancreatic cancer diagnosis is devastating and we are committed to research that aims to change the prognosis for patients. The POLO trial demonstrated that treatment with Lynparza extended time without disease progression or death in patients with germline BRCA-mutated metastatic pancreatic cancer and we are hopeful that we will be able to bring this treatment to patients in Japan soon," Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said.
The Phase III POLO trial showed Lynparza nearly doubled the time patients with gBRCAm metastatic pancreatic cancer lived without disease progression or death to a median of 7.4 months versus 3.8 months on placebo. The safety and tolerability profile of Lynparza in the POLO trial was in line with that observed in previous trials.
Lynparza was approved in the US as a 1st-line maintenance treatment for patients with gBRCAm metastatic pancreatic cancer in December 2019 with ongoing regulatory reviews in the EU and other jurisdictions.
Pancreatic cancer is a deadly cancer with a high unmet medical need. The disease has the lowest survival rate of the most common cancers. Japan has the fifth-highest rate of pancreatic cancer in the world with 43,000 new cases diagnosed in 2018. Pancreatic cancer is the fourth leading cause of cancer death in Japan, resulting in 37,000 deaths in 2018.
Globally, pancreatic cancer is the 11th-most commonly occurring cancer and the seventh leading cause of cancer death. There were approximately 460,000 new cases worldwide in 2018. As there are often no symptoms, or symptoms may be non-specific in the early stages, it is most commonly diagnosed at an incurable stage. Around 80% of pancreatic cancer patients are diagnosed when the disease has metastasized, at which point average survival is less than a year. Despite advances in treatment, few improvements have been made in diagnosis and treatment in the past few decades. Current treatment is surgery (for which approximately only 10-20% of patients are eligible), chemotherapy and radiotherapy, highlighting a critical unmet medical need for more effective treatment options.
POLO is a Phase III randomized, double-blind, placebo-controlled, multi-center trial of Lynparza tablets (300mg twice daily) as maintenance monotherapy versus placebo. The trial randomized 154 patients with gBRCAm metastatic pancreatic cancer whose disease had not progressed on 1st-line platinum-based chemotherapy. Patients were randomized (3:2) to receive Lynparza or placebo until disease progression. The primary endpoint was progression-free survival (PFS) and key secondary endpoints included overall survival (OS), time to second disease progression, overall response rate and health-related quality of life.
BRCA1 and BRCA2 (breast cancer susceptibility genes 1/2) are human genes that produce proteins responsible for repairing damaged DNA and play an important role in maintaining the genetic stability of cells. When either of these genes is mutated, or altered, such that its protein product either is not made or does not function correctly, DNA damage may not be repaired properly, and cells become unstable. As a result, cells are more likely to develop additional genetic alterations that can lead to cancer.
Lynparza (olaparib) is a first-in-class P...
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