Cour Pharmaceuticals’ PBC Treatment Gets FDA Orphan Drug Designation

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The clinical-stage biotechnology company Cour Pharmaceuticals has received an orphan drug designation from FDA for its CNP-104 for treatment of primary biliary cholangitis (PBC). CNP-104 is a biodegradable nanoparticle that encapsulates the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), which is a key autoantigen in PBC, according to Cour in a Jan. 8, 2025 company press release (1).

Orphan drug status is given to treatments for rare diseases, which within the parameters of FDA are characterized as diseases that affect fewer than 200,000 people in the United States (1). The designation allows for various incentives including tax credits for clinical trials, exemption from user fees, and up to seven years of potential market exclusivity following eventual FDA approval.

PBC is an autoimmune liver disease that is a leading cause of liver transplants among women, whom the disease disproportionately impacts (1). It is considered chronic and life-threatening. Typical symptoms include impaired bile flow, or cholestasis, and accumulation of toxic bile acids, which can lead to fibrosis, cirrhosis, and ultimately liver failure, according to Cour.

Other symptoms which Cour said affect everyday quality of life are severe itching, or pruritus, and fatigue (1). CNP-104 was designed to induce tolerance to pathogenic activated PDC-E2 T cells that customarily drive inflammation.

“Receiving [o]rphan [d]rug [d]esignation for CNP-104 underscores its potential to become the first disease-modifying treatment for individuals with PBC,” said Dannielle Appelhans, Cour president and chief executive officer, in the press release (1). “This designation follows our presentation of positive topline data from the Phase IIa clinical trial of CNP-104 in PBC at The Liver Meeting 2024. Notably, in addition to demonstrating favorable T cell responses among treated participants, CNP-104 slowed disease progression, as evidenced by a statistically significant reduction in liver stiffness measured by FibroScan by day 120 of the study period. We are now collaborating with our distinguished clinical advisors and key opinion leaders to advance CNP-104 to the next phase of clinical development.”

In December 2024, the European Medicines Agency’s Committee for Medicinal Products for Human Use recommended an orphan drug approval for another treatment for PBC, Gilead Sciences’ Livdelzi (seladelpar) (2). FDA granted this treatment an accelerated approval in August 2024, and a final European Commission decision is expected in the first quarter of 2025 (3–4).

Cour said that CNP-104 has already received fast track designation from FDA in January 2022, which would make it eligible for accelerated approval and priority review pending the satisfaction of certain criteria (1).

References

1. Cour Pharmaceuticals. Cour Pharmaceuticals Secures FDA Orphan Drug Designation for CNP-104 in Primary Biliary Cholangitis. Press Release. Jan. 8, 2025.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 9–12 December 2024. Press Release. Dec. 13, 2024.
3. Gilead Sciences. Gilead’s Seladelpar Receives Positive CHMP Opinion for Primary Biliary Cholangitis. Press Release. Dec. 13, 2024.
4. Gilead Sciences. Gilead’s Livdelzi (Seladelpar) Granted Accelerated Approval for Primary Biliary Cholangitis by US FDA. Press Release. Aug. 14, 2024.