FDA Approves Ipsen Drug for Rare Liver Disease

The Food and Drug Administration (FDA) has granted accelerated approval to Ipsen's new drug, Iqirvo, for the treatment of primary biliary cholangitis (PBC), a rare liver disease. The approval is based on clinical trial data indicating that Iqirvo improves biochemical markers associated with cholestasis, a condition characterized by the accumulation of bile and toxins in the liver, which leads to inflammation, scarring, and bile duct damage in PBC patients.

The approval of Iqirvo is provisional and contingent on Ipsen's ability to demonstrate through follow-up testing that the improvement in cholestasis markers translates to better survival rates or prevents liver decompensation. This means Ipsen must provide additional evidence of the drug's long-term benefits in forthcoming studies.

Iqirvo now offers a new treatment option for PBC patients, adding to the existing medications, such as Intercept Pharmaceuticals' Ocaliva, which was approved in 2016, marking the first new drug for PBC in over 20 years. The other commonly used medication, ursodeoxycholic acid (UDCA), is effective for some patients but not all, as some experience inadequate responses or intolerable side effects.

Both Ocaliva and Iqirvo have been approved for use in combination with UDCA for adults who do not respond adequately to UDCA alone or as monotherapy for those who cannot tolerate UDCA. Intercept Pharmaceuticals, which entered into an agreement to be acquired by Alfasigma, is working to confirm Ocaliva's 2016 approval with additional data from post-marketing studies and real-world use, with the FDA expected to make a decision by October 15.

Iqirvo and Ocaliva operate through different mechanisms but were both approved based on their ability to reduce alkaline phosphatase (ALP) levels in the blood. Clinical trial results for Iqirvo showed that 51% of participants achieved the primary goal of a biochemical response, compared to only 4% in the placebo group. Furthermore, ALP levels normalized in 15% of those treated with Iqirvo, while none in the placebo group achieved normalization.

Despite its potential benefits, Iqirvo carries several warnings regarding side effects, such as muscle pain, myopathy, rhabdomyolysis, fractures, and drug-induced liver injury. Additionally, there are concerns about potential fetal harm based on animal studies, necessitating caution for pregnant patients. Similarly, Ocaliva also has significant warnings, including a black box warning about hepatic decompensation and liver failure in patients with PBC that has progressed to cirrhosis.

It is worth noting that another potential treatment option for PBC may soon be available. CymaBay Therapeutics, recently acquired by Gilead Sciences for $4.3 billion, has developed a medicine currently under FDA review, with a decision anticipated by mid-August.

PBC affects approximately 100,000 individuals in the United States and is known for causing progressive liver damage, severe fatigue, and intense itching. Ipsen's licensing of Iqirvo from Genfit three years ago included a milestone payment of around 50 million euros, which is now due following the drug's first commercial sale.