FDA Grants Orphan Status to Targeted Treatment for Pancreatic Cancer

Olaparib (Lynparza, AstraZeneca and Merck), which is being investigated for the treatment of prostate cancer, receives its fourth orphan drug designation from the FDA.

The investigational therapy olaparib (Lynparza, AstraZeneca and Merck) has been granted Orphan Drug Designation (ODD) by the FDA for the treatment of pancreatic cancer, according to an AstraZeneca press release.

There is an unmet clinical need for additional pancreatic cancer therapies, especially for patients with metastatic disease. Pancreatic cancer accounts for approximately 3% of all cancers in the United States and the 5-year survival rates for patients with the disease are 8.5%, according to the American Cancer Society. The FDA grants ODD status to medications intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 individuals in the United States.

Olaparib is currently being evaluated in the ongoing phase 3 POLO trial, which is testing the safety and efficacy of olaparib tablets (300 mg twice daily) as a maintenance monotherapy compared with placebo. The trial includes 145 patients with germline BRCA-mutated metastatic pancreatic cancer whose disease has not progressed following first-line platinum-based chemotherapy, with a primary endpoint of progression-free survival.

Olaparib is a first-in-class PARP inhibitor and the first targeted treatment to potentially exploit DNA damage response pathway deficiencies, such as BRCA mutations, to preferentially kill cancer cells, according to AstraZeneca. Olaparib is also being investigated in a range of DDR-deficient tumor types.

“Pancreatic cancer is a relatively less common, but life-threatening, form of cancer,” Roy Baynes, senior vice president and head of global clinical development and chief medical officer at MSD Research Laboratories, said in the press release. “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 for more patients in need.”

In October 2013, olaparib received ODD status for the treatment of ovarian cancer. In early 2018, the treatment was granted an amended ODD status to include both fallopian tube and primary peritoneal cancer following its expanded approval in August 2017, making this the fourth time olaparib has been granted ODD.

Olaparib is being jointly developed and commercialized by AstraZeneca and Merck.


US FDA grants Lynparza Orphan Drug Designation for pancreatic cancer [news release]. AstraZeneca’s website. https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2018/us-fda-grants-lynparza-orphan-drug-designation-for-pancreatic-cancer-16102018.html. Accessed October 16, 2018.