BERWYN, PA — Panavance Therapeutics Inc. announced the U.S. Food and Drug Administration (FDA) recently granted Orphan Drug Designation to GP-2250 for the treatment of all forms of pancreatic cancer.
GP-2250 is the Company’s broadly active, tumor cell-selective cancer therapeutic with a unique mechanism of action that, as demonstrated in preclinical research, suppresses cancer cells by disrupting their energy metabolism—bringing about cancer cell death. GP-2250 has demonstrated a promising drug safety profile in both preclinical studies and early results seen to date in the Phase 1 pancreatic cancer clinical study.
“We are dedicated to improving therapeutic outcomes for cancer patients and believe GP-2250 has the potential to positively impact the treatment landscape for pancreatic cancer. Receiving Orphan Drug Designation from the FDA bolsters our efforts and confidence as we rapidly advance this important clinical program to address an area of significant unmet need. We continue to make solid progress on the clinical front and are dedicated to advancing this program as quickly and efficiently as possible,” commented Greg Bosch, Chairman and CEO of Panavance Therapeutics.
Pancreatic cancer has the highest mortality rate of all major cancers and is the seventh leading cause of cancer death globally with survival only 1% for Stage IV patients after 5 years. Most pancreatic cancer begins in the cells that line the ducts of the pancreas. This type of cancer, called pancreatic ductal adenocarcinoma (PDAC), is a highly aggressive lethal malignancy due to the lack of early diagnosis and limited response to treatments. PDAC is the most prevalent type of pancreatic neoplasm, accounting for more than 90% of pancreatic cancer cases. Current treatment options include surgically removing the pancreas, radiation, and chemotherapy.
Panavance is currently evaluating GP-2250, in combination with gemcitabine, in a Phase 1 first in human, dose-escalation trial in patients with advanced or metastatic pancreatic cancer previously treated with FOLFIRINOX but never exposed to gemcitabine. Additionally, the Company has conducted extensive preclinical studies evaluating GP-2250 for the treatment of numerous other cancers including ovarian and endometrial carcinomas.
The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets certain cost recovery provisions. Orphan drug designation qualifies sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees, and potential seven years of market exclusivity after approval.1
1(May 2022). Developing Products for Rare Diseases & Conditions. U.S. Food and Drug Administration. https://www.fda.gov/industry/developing-products-rare-diseases-conditions
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