Elafibranor

FDA grants Cour Pharmaceuticals orphan drug designation for CNP-104, a nanoparticle treatment for primary biliary cholangitis (PBC), following positive phase 2a trial results. CNP-104 shows potential to modify disease progression, with significant liver health improvements. This designation supports its development amidst a changing PBC treatment landscape, offering incentives like tax credits and market exclusivity.

COUR Pharmaceuticals' CNP-104 received FDA Orphan Drug Designation for PBC treatment, following positive phase 2a trial results showing reduced liver stiffness and favorable T cell responses. CNP-104, targeting PBC's root cause, aims to induce tolerance to pathogenic T-cells, with no significant safety risks observed in trials.

In 2024, FDA's CDER approved 50 new drugs, including 32 NCEs and 18 NBEs, with small molecules and antibody-based drugs dominating. First-in-class drugs accounted for 44% of approvals, with notable approvals like Rezdiffra for NASH and Voranigo for brain tumors. Nucleic acid and peptide-based drugs made up 10% of approvals, including Rytelo for MDS. FDA also approved 8 cell and gene therapies, marking a record and introducing first treatments for several diseases.

Ocaliva, once a promising treatment for primary biliary cholangitis (PBC), faces FDA setbacks due to safety concerns, including serious liver injury. Despite initial approval in 2016, its potential has narrowed, competing with newer, safer drugs for PBC.

As of Dec 5, 2024, FDA approved 44 novel drugs, including 9 oncology treatments, 3 cardiovascular therapies, and 3 diagnostic/imaging agents. Notable approvals include Bizengri for lung/pancreatic cancers, Attruby for transthyretin-mediated amyloidosis, and Ziihera for HER2-positive biliary tract cancer. Oncology dominated with first-in-class treatments and precision medicine advancements.

The FDA identified additional safety concerns with Ocaliva, used to treat primary biliary cholangitis, including serious liver injury in non-cirrhotic patients. Since 2021, 20 more liver-related events were reported. The FDA advises frequent liver tests and discontinuation if disease progresses or if the drug is ineffective. Ocaliva, once a blockbuster prospect, faces setbacks with FDA rejections and competition from new drugs.

Approval of ursodeoxycholic acid (UDCA) in 1997 significantly reduced liver transplant and death risks for primary biliary cholangitis (PBC), but up to 40% of patients do not sufficiently respond. Off-label use of fibrates and the 2016 approval of obeticholic acid (Ocaliva) as a second-line treatment provided additional options, though Ocaliva has downsides like increased pruritus. New PPAR agonists, seladelpar and elafibranor, have been approved for PBC, showing potential benefits but with unknown long-term risks.

Advanz Pharma's Ocaliva (obeticholic acid) for PBC loses EU market access as EC revokes conditional marketing authorisation, leaving limited second-line options for patients. Advanz CEO criticizes court's ruling for not assessing patient impact. Ocaliva remains available in the US under accelerated approval.

Ipsen presents Iqirvo data at AASLD 2024, showing sustained efficacy and safety in PBC patients over 3 years, with improvements in pruritus, fatigue, and sleep. Iqirvo is approved in the U.S., E.U., and U.K. for PBC treatment.