Drug rivals face off in PBC after FDA hands down 2 new approvals, 1 advisory snub
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The long-stagnant primary biliary cholangitis drug market has seen renewed attention in the past 3 months with the addition of two FDA-approved therapy options as well as a costly ruling from an FDA advisory committee.
Although ursodeoxycholic acid (UDCA) remains the only first-line treatment for PBC, the number of patients who fail to adequately respond with UDCA or cannot tolerate its use has continue to drive demand for second-line options.
The introduction of two oral peroxisome proliferator-activated receptor therapies, Iqirvo (elafibranor, Ipsen/Genfit) and Livdelzi (seladelpar, Gilead), serve as the first significant change in the PBC therapy landscape since the FDA granted Ocaliva (obeticholic acid, Intercept Pharmaceuticals) accelerated approval for PBC in 2016.
However, following an unfavorable meeting of the FDA’s Gastrointestinal Drug Advisory Committee on Sept. 13, long-term safety and efficacy data for Ocaliva may not support its full approval and keep the drug from securing its position in the market against other contenders in the pipeline.
Healio has closely followed updates for PBC therapies throughout the summer, reporting on news that broke major headlines and sitting down with experts to get their insights on this evolving landscape. Want to see what you missed? We tallied up the pivotal FDA moves expected to change the treatment paradigm for PBC in 2024 and beyond.
FDA approves Ipsen’s Iqirvo ‘to address unmet need’ in primary biliary cholangitis
The FDA granted accelerated approval to Ipsen Pharma’s Iqirvo 80 mg, a first-in-class oral, once-daily peroxisome proliferator-activated receptor agonist for the treatment of primary biliary cholangitis, according to a company release.
Originally given breakthrough therapy designation in 2019, Iqirvo (elafibranor, Ipsen/Genfit), is now indicated as second-line therapy in combination with ursodeoxycholic acid (UDCA) for adult patients with inadequate response to UDCA, or as monotherapy in those who are intolerant to UDCA. Read more
FDA approves Gilead’s Livdelzi for treatment of primary biliary cholangitis
Gilead’s Livdelzi, a selective peroxisome proliferator-activated receptor delta agonist, has received accelerated FDA approval for the treatment of primary biliary cholangitis, according to a company release.
Livdelzi (seladelpar) was approved for use in combination with ursodeoxycholic acid (UDCA) in adult patients who did not adequately respond to UDCA or as monotherapy for those who are intolerant to UDCA. Read more
FDA advisory panel votes against Ocaliva for PBC citing ‘concern for real possible harm’
An FDA advisory committee voted against approval for obeticholic acid in primary biliary cholangitis without cirrhosis or compensated cirrhosis with portal hypertension, citing “concern for real possible harm”.
The Gastrointestinal Drug Advisory Committee voted 13 to 1 with no abstentions that the benefits of obeticholic acid (Ocaliva, Intercept Pharmaceuticals) on clinical outcomes in patients with PBC could not be verified with available data from the postmarketing requirement confirmatory trial, 747-302 and the observational study, 747-405. Read more
A new era for PBC treatment: Expert insights on existing, emerging therapies
Primary biliary cholangitis poses a significant challenge in hepatology, necessitating effective treatment strategies to improve patient outcomes and quality of life.
In a recent interview, James Boyer, MD, FRCP, Ensign Professor of Medicine and emeritus director of the Liver Center at Yale School of Medicine, provided valuable insights into the evolving landscape of primary biliary cholangitis (PBC) therapeutics, emphasizing both established treatments and promising innovations. Read more
Q&A: ‘Make them explain’: How a patient’s self-advocacy finally led to a PBC diagnosis
When Tiffany McLellan was 18 years old, she was diagnosed with an ulcer in her digestive tract. She found relief with antacids for almost 2 years, but after her son was born, she started experiencing digestive issues and pain again.
Doctors told her it was irritable bowel syndrome, but they didn’t provide proof, nor would they do a colonoscopy since she was too young. McLellan wasn’t convinced, but she went along with the IBS diagnosis for almost 18 years. Read more