Elafibranor, sold under the brand name Iqirvo, is a

medication Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to medical diagnosis, diagnose, cure, treat, or preventive medicine, prevent disease. Drug therapy (pharmaco ...

used for the treatment of

primary biliary cholangitis Primary biliary cholangitis (PBC), previously known as primary biliary cirrhosis, is an autoimmune disease of the liver. It results from a slow, progressive destruction of the small bile ducts of the liver, causing bile and other toxins to buil ...

. Elafibranor is a dual PPARα/δ agonist. Elafibranor and its main active metabolite GFT1007 are peroxisome proliferator-activated receptor (PPAR) agonists, both of which activate PPAR-alpha, PPAR-gamma, and PPAR-delta in vitro. In June 2024, the US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) granted accelerated approval to elafibranor. The FDA considers it to be a

first-in-class medication A first-in-class medication is a prototype drug that uses a "new and unique mechanism of action" to treat a particular medical condition. While the Food and Drug Administration's Center for Drug Evaluation and Research tracks first-in-class medic ...

Medical uses

Elafibranor is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

for the treatment of

in combination with

ursodeoxycholic acid Ursodeoxycholic acid (UDCA), also known as ursodiol, is a secondary bile acid, produced in humans and most other species from metabolism by intestinal bacteria. It is synthesized in the liver in some species, and was first identified in bile o ...

in adults who have an inadequate response to ursodeoxycholic acid, or as monotherapy in people unable to tolerate ursodeoxycholic acid.

Adverse effects

The most common adverse reactions include weight gain, diarrhea, abdominal pain, nausea, vomiting, arthralgia, constipation, muscle injury, fracture, gastroesophageal reflux disease, dry mouth, weight loss, and rash.

History

In 2019, the US

(FDA) granted elafibranor

breakthrough therapy Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "b ...

designation, based on phase II data, for the treatment of

in adults 18 and older with inadequate response to ursodeoxycholic acid (UDCA). The designation was granted to Genfit. In June 2024, the US FDA granted accelerated approval to elafibranor. The approval was based on positive phase III ELATIVE trial data. The designation was granted to

Ipsen Ipsen is a French biopharmaceutical company headquartered in Paris, France, with a focus on drug development and commercialization in three therapeutic areas: oncology, rare diseases and neuroscience. Ipsen is one of the world's top 15 biopha ...

Society and culture

Legal status

In July 2024, the

Committee for Medicinal Products for Human Use The Committee for Medicinal Products for Human Use (CHMP), formerly known as the Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regar ...

(CHMP) of the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products ...

adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Iqirvo, intended for the treatment of primary biliary cholangitis (PBC). The applicant for this medicinal product is Ipsen Pharma. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Elafibranor was authorized for medical use in the European Union in September 2024. In October 2024, the National Institute for Health and Care Excellence (NICE) adopted a recommendation for elafibranor for the treatment of adults with the

based results from the phase 3 ELATIVE trial, in which 51% of patients receiving elafibranor in combination with

achieved a cholestasis response at week 52, compared to 4% of those in the placebo plus ursodeoxycholic acid group.

Names

Elafibranor is the international nonproprietary name. Elafibranor is sold under the brand name Iqirvo.

Research

Elafibranor is being studied and developed by Genfit for the treatment of endocrine and metabolic diseases such as

type 2 diabetes Type 2 diabetes (T2D), formerly known as adult-onset diabetes, is a form of diabetes mellitus that is characterized by high blood sugar, insulin resistance, and relative lack of insulin. Common symptoms include increased thirst, frequent ...

dyslipidemia Dyslipidemia is a metabolic disorder characterized by abnormally high or low amounts of any or all lipids (e.g. fats, triglycerides, cholesterol, phospholipids) or lipoproteins in the blood. Dyslipidemia is a risk factor for the development of ...

, and metabolic dysfunction–associated steatohepatitis.

References

{{Authority control PPAR agonists Orphan drugs Carboxylic acids Thioethers Aromatic ketones Aromatic ethers