FDA Grants Orphan Drug Status to PEP-Therapy's PEP-010 for Pancreatic Cancer

PEP-Therapy, a biotechnology firm based in Paris, is making strides in the field of oncology with the development of pioneering peptide therapeutics. On March 17, 2025, the company announced that its flagship product, PEP-010, has received the Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its use in treating pancreatic cancer.

PEP-010 is a first-in-class bifunctional therapeutic peptide being evaluated in an ongoing Phase Ib clinical trial. This trial aims to assess the drug’s safety, tolerability, pharmacokinetics, and initial anti-tumor activity as it is used alongside chemotherapy agents like paclitaxel or gemcitabine, particularly in patients battling pancreatic cancer.

Dr. Hatem Azim, Chief Medical Officer at PEP-Therapy, emphasized the significance of this designation, noting that it marks a critical milestone in addressing the pressing need for new treatments for pancreatic cancer patients. These individuals currently face limited treatment options, and the rising incidence and mortality rates of this disease highlight the urgent need for innovative therapies. The Orphan Drug Designation, along with promising early data from the Phase I study, bolsters the potential of PEP-010 as a novel therapy.

The CEO and co-founder of PEP-Therapy, Antoine Prestat, also highlighted the importance of obtaining the FDA’s Orphan Drug Designation. He expressed optimism about the impact this will have on accelerating the development of PEP-010, ultimately aiming to provide an innovative solution for difficult-to-treat cancers. Prestat looks forward to sharing updated clinical data as the trials progress.

The FDA's Office of Orphan Products Development awards the Orphan Drug Designation to new drugs or biological products aimed at diagnosing, preventing, or treating rare diseases that affect fewer than 200,000 people in the U.S. This designation brings several development and commercial advantages, including tax credits for eligible clinical trials, waivers of major regulatory fees, and the possibility of obtaining up to seven years of market exclusivity after the drug is approved.

In addition to PEP-010, PEP-Therapy is building a pipeline of peptide-based therapeutic products targeting various oncology applications. Founded in 2014, the company draws on research from Sorbonne University and Institut Curie and has garnered support from international investors such as Seventure Partners, CapHorn, i&i Prague, Italian Angels for Growth, among others.

PEP-010 works by entering cells and disrupting the interaction between two key proteins in the apoptotic pathway: Caspase-9 and PP2A. By doing so, the peptide helps restore normal apoptosis in cancer cells. The agent has demonstrated promising safety and efficacy signals in the CLEVer-PEPtide Phase Ia dose-escalation trial and is currently being tested in a Phase Ib trial for patients with metastatic pancreatic ductal adenocarcinoma and advanced or metastatic ovarian cancer. Four recruitment sites in France are actively participating in these studies.

Pancreatic cancer poses a significant health challenge, being the fourth leading cause of cancer-related deaths in the United States. With roughly 66,000 new cases and 51,000 deaths each year, mortality rates continue to rise. Surgical resection remains the only potentially curative intervention, yet over 80% of patients are diagnosed with unresectable disease. Even with resection and adjuvant therapy, survival averages just 3-4 years, and outcomes for advanced cases are dire, with only 3% of patients surviving beyond one year. Given the limited progress in systemic treatment options over the past decades, there is a pressing need for innovative therapies that could improve survival outcomes while maintaining quality of life for patients.