Cour Pharmaceuticals snags orphan drug designation for CNP-104 for the treatment of PBC
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The FDA has granted Cour Pharmaceuticals orphan drug designation for CNP-104, a biodegradable nanoparticle in development for the treatment of primary biliary cholangitis, the company announced in a press release.
According to Cour, CNP-104 initiates tolerance to pathogenic activated pyruvate dehydrogenase complex-E2 T-cells that “drive inflammation in bile ducts, leading to improvement in clinical outcomes of liver health.”
The investigational therapy was granted fast track designation by the FDA in 2022, bringing it one step closer to receiving accelerated FDA approval and priority review if specific criteria are met.
“Receiving orphan drug designation for CNP-104 underscores its potential to become the first disease-modifying treatment for individuals with PBC,” Dannielle Appelhans, Cour’s president and CEO, said in the release, noting the designation follows presentation of positive topline data from the phase 2a clinical trial of CNP-104 in PBC at The Liver Meeting 2024.
She continued, “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.”
CNP-104’s orphan drug designation comes after the long-stagnant PBC treatment landscape saw two new drugs — Iqirvo (elafibranor, Ipsen/Genfit) and Livdelzi (seladelpar, Gilead) — receive accelerated approval for PBC in 2024. Ocaliva (obeticholic acid, Intercept Pharmaceuticals), once thought to be a strong contender after receiving accelerated approval in 2016, had many setbacks last year, including an FDA advisory committee vote against full approval. The agency officially rejected full approval 1 month later.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
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