Tuesday, July 16, 2024

Biliary Cholangitis: FDA Approves Iqirvo, First-in-Class PPAR Therapy

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Biliary Cholangitis has a new treatment option as the US Food and Drug Administration (FDA) has granted accelerated approval for Iqirvo (elafibranor; Ipsen). This approval applies to its use in combination with ursodeoxycholic acid (UDCA) in adults who do not respond adequately to UDCA or as monotherapy in patients unable to tolerate UDCA.

PBC is a rare, chronic cholestatic liver disease that progressively destroys the interlobular bile ducts, leading to cholestasis and liver fibrosis. If left untreated, PBC can advance to cirrhosis, necessitating liver transplantation and, in some cases, causing premature death. The disease significantly impacts the quality of life, with patients often suffering from severe fatigue and pruritus.

Biliary Cholangitis: FDA Approves Iqirvo, First New Treatment in Nearly a Decade

Iqirvo is the first new drug approved in nearly a decade for the treatment of PBC. It is an oral dual peroxisome proliferator–activated receptor (PPAR) alpha and delta agonist. The accelerated approval was based on data from the phase 3 ELATIVE trial, which was published in The New England Journal of Medicine.

The ELATIVE trial randomly assigned patients with PBC who had an inadequate response to or unacceptable side effects with UDCA to receive either once-daily elafibranor (80 mg) or a placebo. The primary endpoint was a biochemical response, defined as an alkaline phosphatase (ALP) level less than 1.67 times the upper limit of the normal range, with a reduction of at least 15% from baseline, as well as normal total bilirubin levels.

Among 161 patients, a biochemical response was observed in 55 of 108 (51%) who received elafibranor compared to 2 of 53 (4%) who received placebo. By week 52, the ALP level normalized in 15% of patients in the elafibranor group, while none of the patients in the placebo group achieved normalization.

Biliary Cholangitis

Biliary Cholangitis: Continued Approval of Iqirvo Dependent on Confirmatory Trials for Survival and Liver Decompensation Benefits

In the news release announcing the approval of Iqirvo, the company highlighted that improvement in survival and prevention of liver decompensation events have not yet been demonstrated. Continued approval for PBC may be contingent upon verification and description of clinical benefit in confirmatory trials.

The most common adverse effects reported in ≥10% of study participants included weight gain, abdominal pain, diarrhea, nausea, and vomiting. Iqirvo is not recommended for individuals who have or develop decompensated cirrhosis. Detailed prescribing information is available online.

Kris Kowdley, MD, director of the Liver Institute Northwest in Seattle, Washington, and a primary investigator on the ELATIVE study, stated in the news release, “The data show that Iqirvo is an effective second-line treatment for patients with PBC with favorable benefit and risk data.” He emphasized that the approval of Iqirvo “will allow healthcare providers in the US to address an unmet need with the potential to significantly reduce ALP levels for our patients with PBC.”

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The FDA’s accelerated approval of Iqirvo represents a significant advancement in the treatment options available for patients with primary biliary cholangitis. By offering a new therapeutic option, Iqirvo addresses a critical gap in the management of this chronic and debilitating disease. The ongoing and future confirmatory trials will be essential in solidifying its role in improving patient outcomes and further validating its clinical benefits.

As healthcare providers begin to integrate Iqirvo into treatment protocols, the focus will remain on monitoring its efficacy and safety in real-world settings. The potential to alleviate the symptoms and slow the progression of PBC offers hope to patients and their families, highlighting the importance of continuous innovation and research in the field of hepatology.

Resource: İpsen, June 10, 2024

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