Halozyme Treatment Wins FDA Orphan Status For Pancreatic Cancer

Biopharmaceutical company Halozyme Therapeutics reported that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its PEGylated recombinant human hyaluronidase (PEGPH20) as treatment for pancreatic cancer.

PEGPH20 is being developed for the systemic treatment of tumors that accumulate hyaluronan. The company is currently conducting a Phase 2 study for the drug in combination with gemcitabine and nab-paclitaxel (Abraxane) in patients with metastatic pancreatic cancer. In September, the company reported that the FDA granted Fast track designation to the program evaluating PEGPH29 in combination with other therapies in this indication.

Dr. Helen Torley, President and CEO of Halozyme, said that the orphan designation is a major step for the company’s clinical development program for PEGPH20. “This designation follows the Fast Track designation and allows us to collaborate more closely with the FDA to facilitate development of PEGPH20 and is part of our core strategy to bring this therapy to patients as rapidly as possible.”

The FDA’s orphan drug program awards orphan status to drugs, biologics, and therapies that target rare diseases affecting fewer than 200,000 people in the U.S. Should PEGPH20 receive approval for its orphan designated indication, Halozyme will receive a seven-year marketing exclusivity period in the U.S. The company will also receive other incentives, including exemption from certain regulatory fees.

Only last month, Halozyme reported that the FDA has lifted its clinical hold on the Phase 1B/2 study of the drug in combination with a modified FOLFIRINOX chemotherapy (mFOLFIRINOX) indicated for patients with metastatic pancreatic adenocarcinoma. The clinical hold was enforced due to concerns that the drug caused thromboembolic events among patients. The company resumed the study under a revised protocol and has reopened enrollment of patients for the trial. Approximately 170 patients will be enrolled in the study, which is being sponsored by cancer research cooperative group SWOG.