Dive Brief:
- The Food and Drug Administration on Monday cleared a new treatment option for the rare liver disease primary biliary cholangitis, granting an accelerated approval to Ipsen’s drug Iqirvo.
- The agency’s OK was based on clinical trial data showing treatment with Iqirvo improved certain biochemical markers for cholestasis, or the accumulation in the liver of bile and toxins. In people with primary biliary cholangitis, or PBC, this build-up and its associated inflammation causes scarring and damages bile ducts.
- Iqirvo’s place on the market is contingent on Ipsen proving in follow-up testing that changes in cholestasis markers lead to improved survival or prevent liver decompensation.
Dive Insight:
Iqirvo’s approval gives people with PBC another treatment option beyond Intercept Pharmaceuticals’ Ocaliva, which was approved in 2016 as the first new drug for the disease in two decades.
The other approved medicine, ursodeoxycholic acid or UDCA, is effective in some patients, but others don’t respond well or are unable to tolerate the drug’s side effects. The FDA’s approvals for Ocaliva in 2016 and for Iqirvo Monday specify use in combination with UDCA for adults who have an inadequate response to that medicine, or as monotherapy in those people who can’t take UDCA.
Intercept, which last year agreed to sell to the Italian company Alfasigma, is seeking to confirm Ocaliva’s 2016 approval with follow-up data from post-marketing studies and real-world use. An FDA decision on full approval is expected by Oct. 15.
Iqirvo and Ocaliva work differently, but both were cleared based on their ability to reduce levels of an enzyme called ALP in the blood. Data from the trial supporting Iqirvo’s approval showed that 51% of treated study participants met the main goal of a biochemical response, compared to only 4% of those given a placebo. ALP levels were normalized in 15% of people who received Iqirvo, versus none in the placebo group.
Iqirvo’s labeling warns of a wide range of side effects, including muscle pain, myopathy, rhabdomyolysis, fractures and drug-induced liver injury. Based on animal data, the label also cautions of the potential for fetal harm in people who are pregnant.
Ocaliva also comes with significant cautions, including a black box warning for hepatic decompensation and failure in people with PBC that’s led to cirrhosis.
The two drugs may soon be joined by another therapy from CymaBay Therapeutics, which was recently acquired by Gilead Sciences in a $4.3 billion deal. That medicine is currently under FDA review, with a decision set for mid-August.
PBC is thought to affect about 100,000 people in the U.S. It causes progressive damage and typically is associated with severe fatigue and intense itching.
Ipsen licensed Iqirvo three years ago from Genfit, which is owed about 50 million euros in a milestone payment timed to the drug’s first commercial sale.
