| Drug Name |
IQIRVO® (elafibranor) |
| Active Ingredient |
elafibranor |
| Date of Approval |
06/10/24 |
| Manufacturer |
Ipsen Biopharmaceuticals, Inc. |
| Approval Pathways and Indications |
Approval Pathway: Accelerated Approval - 2024; NDA - December 2023
Indication: IQIRVO® is a peroxisome proliferator-activated receptor (PPAR) agonist indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.
*This indication is approved under accelerated approval based on reduction of alkaline phosphatase (ALP). Improvement in survival or prevention of liver decompensation events have not been demonstrated. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). |
| Therapeutic Class |
PPAR Agonist |
| Formulation |
Tablets, for Oral Use |
| MoA |
Elafibranor and its main active metabolite, GFT1007, are PPAR agonists, both of which activate PPAR-alpha, PPAR-gamma, and PPAR-delta in vitro. However, the mechanism by which elafibranor exerts its therapeutic effects in patients with PBC is not well understood. Pharmacological activity that is potentially relevant to therapeutic effects includes inhibition of bile acid synthesis through
activation of PPAR-alpha and PPAR-delta. |
| Dosing and Administration |
The recommended dosage is 80 mg orally once daily with or without food.
Before treatment, evaluate for muscle pain or myopathy, and/or verify that females of reproductive potential are not pregnant. |
| Administered by self or by HCP |
Administered by self |
| Place in Therapy |
PBC is a rare, autoimmune, cholestatic liver disease where a build-up of bile and toxins (cholestasis) and chronic inflammation causes irreversible fibrosis (scarring) of the liver and destruction of the bile ducts. Impacting approximately 100,000 people in the U.S., the majority being women, PBC is a lifelong condition that can worsen over time if not effectively treated, leading to liver transplant and in some cases, premature death. PBC also affects day-to-day life, with people most commonly experiencing severe fatigue symptoms and debilitating itch (pruritus).
“For a significant number of people living with PBC, available treatments do not control the condition and may exacerbate symptoms of PBC. Left unmanaged, PBC can progress, leading to liver failure and in some cases, the need for a liver transplant,” said Christelle Huguet, Executive Vice President, Head of Research and Development at Ipsen. “IQIRVO® demonstrated statistically significant improvements in biochemical response compared to UDCA alone. IQIRVO® is, therefore a much-needed treatment option and the first new medicine for PBC in nearly a decade.” |
| Expected Market Launch Date |
The information is currently unavailable, though some sources suggest that the drug is already available for prescription. |
| New Molecular Entity (NME) or Existing Formulation |
NME |
| Expected Cost |
The estimated price of IQIRVO® is $11,500 for a month's supply. |
| Product Discontinuation |
Previously, IQIRVO® failed as a treatment for the fatty liver disease metabolic dysfunction-associated steatohepatitis (MASH). |
| Clinical Trials |
The accelerated approval of IQIRVO® is based on data from the Phase III ELATIVE trial published in the New England Journal of Medicine. The ELATIVE trial demonstrated that 13 times more patients achieved the composite primary endpoint of biochemical response when treated with IQIRVO® plus UDCA (n=108) versus placebo plus UDCA (=53) (respectively 51% versus 4% for a 47% treatment difference). ALP is a biochemical marker and is used as a surrogate endpoint in PBC trials. Secondary endpoints showed normalization in ALP levels in only IQIRVO®-treated patients (15% for IQIRVO® plus UDCA (n=108) versus 0% for placebo plus UDCA (n=53). Most patients (95%) received study treatment (IQIRVO® or placebo) in combination with UDCA.
The most common adverse reactions with IQIRVO® reported in ≥10% of study participants were weight gain, abdominal pain, diarrhea, nausea, and vomiting. Some study participants treated with Iqirvo experienced myalgia, myopathy and rhabdomyolysis; fractures; adverse effects on fetal and newborn development; drug-induced liver injury; hypersensitivity reactions; or biliary obstruction. |
| |
Ipsen’s Iqirvo® receives U.S. FDA accelerated approval as a first-in-class PPAR treatment for primary biliary cholangitis. United States. (2024, June 11). https://www.ipsen.com/
us/press-releases/
ipsens-iqirvo-receives-u-s-fda-accelerated-approval-
as-a-first-in-class-ppar-treatment-for-primary-biliary-cholangitis/#:~:text=Left%
20unmanaged%2C%20PBC%20can%20progress,respons
e%20compared%20to%20UDCA%20alone. |