AstraZeneca and Merck (NYSE:MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) for LYNPARZA for the treatment of pancreatic cancer. LYNPARZA is currently being investigated as maintenance therapy in patients with germline BRCA-mutated (gBRCAm) metastatic pancreatic cancer whose disease has not progressed following first-line platinum-based chemotherapy.
Pancreatic cancer is a rare, life-threatening disease that accounts for about 3 percent of all cancers in the U.S. 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 U.S. at 8.5 percent.
Sean Bohen, executive vice president, Global Medicines Development, and chief medical officer at AstraZeneca, 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.”
Dr. Roy Baynes, senior vice president and head of Global Clinical Development, chief medical officer, Merck Research Laboratories, said, “Pancreatic cancer is a relatively less common, but life-threatening, form of cancer. The FDA Orphan Drug Designation reinforces the importance of our collaboration in bringing LYNPARZA to patients in need.”
This is the fourth ODD in the U.S. for LYNPARZA. 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 U.S. 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 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 U.S. Through the ODD program, the FDA provides incentives for pharmaceutical companies to develop products for rare diseases.
The use of LYNPARZA in pancreatic cancer is being assessed in the ongoing Phase 3 POLO trial, which is investigating LYNPARZA as maintenance monotherapy versus placebo in patients with gBRCAm metastatic pancreatic cancer whose disease has not progressed following first-line platinum-based chemotherapy. Results from the POLO trial are expected in the first half of 2019.
Important Safety Information
There are no contraindications for LYNPARZA.
Warnings and Precautions
Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML): Occurred in <1.5% of patients exposed to LYNPARZA monotherapy, and the majority of events had a fatal outcome. The duration of therapy in patients who developed secondary MDS/AML varied from <6 months to >2 years. All of these patients had previous chemotherapy with platinum agents and/or other DNA-damaging agents, including radiotherapy, and some also had a history of more than one primary malignancy or of bone marrow dysplasia.
Do not start LYNPARZA until patients have recovered from hematological toxicity caused by previous chemotherapy (≤Grade 1). Monitor complete blood count for cytopenia at baseline and monthly thereafter for clinically significant changes during treatment. For prolonged hematological toxicities, interrupt LYNPARZA and monitor blood count weekly until recovery.
If the levels have not recovered to Grade 1 or less after 4 weeks, refer the patient to a hematologist for further investigations, including bone marrow analysis and blood sample for cytogenetics. Discontinue LYNPARZA if MDS/AML is confirmed.
Pneumonitis: Occurred in <1% of patients exposed to LYNPARZA, and some cases were fatal. If patients present with new or worsening respiratory symptoms such as dyspnea, cough, and fever, or a radiological abnormality occurs, interrupt LYNPARZA treatment and initiate prompt investigation. Discontinue LYNPARZA if pneumonitis is confirmed and treat patient appropriately.