The U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for elafibranor, an investigational therapeutic for the treatment of primary biliary cholangitis (PBC) developed by Ipsen and GENFIT, according to a recently published press release.
The FDA is a government agency that regulates the registration of food, drugs, cosmetics, and medical products in the U.S. Submitting an NDA is part of the registration process. The NDA provides enough information for the FDA to decide whether a new drug is safe and effective for its proposed use. The application was granted priority review, and the FDA set a review date of June 10, 2024.
Christelle Huguet, EVP and Head of Research & Development at Ipsen, stated elafibranor has the potential to change the management of PBC and offer a new second-line treatment choice for patients.
Learn more about PBC therapies
The European Medicines Agency (EMA) has also approved a Marketing Authorization Application (MAA) for elafibranor. The EMA’s Committee for Medicinal Products for Human Use started reviewing the submitted documents on Oct. 26, and it is also being reviewed by the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA).
The novel medication has been investigated in a clinical trial called ELATIVE. This trial enrolled 161 patients with PBC who did not respond well to ursodeoxycholic acid, the current standard of care. Study participants were randomly assigned to groups receiving elafibranor or placebo (an inactive substance). Both patients and doctors were unaware of who was receiving the experimental drug.
According to the results, the therapy may slow down disease progression and reduce the intensity of itching. It was overall safe and well-tolerated by patients. However, less than 10% of study participants experienced adverse effects, such as abdominal pain, diarrhea, nausea and vomiting.
