Promising Liver Disease Drugs in the Pipeline for 2021 to 2022

Three notable medications to treat liver diseases are expected to come online in the third quarter of 2021 and in 2022, according to PBM OptumRx.

One key drug to watch for 2022 is Intercept Pharmaceuticals’ obeticholic acid for nonalcoholic steatohepatitis (NASH), Savitha Vivian, senior vice president of Clinical and Formulary Services at OptumRx, told Managed Healthcare Executive®.

NASH is a very common chronic liver condition with no FDA approved treatments, Vivian noted. Obeticholic acid was originally rejected by the FDA in 2020 when the agency asked Intercept for more data, and the agency’s recent Complete Response Letter “determined that the predicted benefit of OCA based on a surrogate histopathologic endpoint remains uncertain and does not sufficiently outweigh the potential risks to support accelerated approval for the treatment of patients with liver fibrosis due to NASH,” Intercept said in a news release.

The FDA recommends that Intercept submit additional post-interim analysis efficacy and safety data from its ongoing REGENERATE study in support of potential accelerated approval.

“It is possible that we will get more data and a potential FDA resubmission in 2022, but the approval decision is likely in the second half of 2022,” Vivian said.

Meanwhile, two treatments for rare liver diseases are expected to be available this fall or later in 2021, according to OptumRx’s Promising Drugs in the Pipeline report,

In late July, the Food and Drug Administration approved Albireo Pharma’s Bylvay (odevixibat), the first drug approved for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC).

Albireo said in a news release that it is launching Bylvay immediately to “accelerate availability for the patients and families impacted by PFIC,” a rare and devastating disorder affecting young children that causes progressive, life-threatening liver disease.

While there is a treatable population of about 600 people with PFIC, there is a “high unmet need” for effective treatments for the disease, Vivian explained.

“PFIC is typically unresponsive to conventional treatments that are used off-label, like ursodeoxycholic acid, antihistamines, or rifampicin. The only proven effective treatments are surgical interventions and transplantation,” she said.

The estimated average cost for Bylvay is $385,000 annually, according to Vivian.

Meanwhile, Miriam Pharmaceuticals’ maralixibat, an oral apical sodium dependent bile acid transporter (ASBT) inhibitor to treat cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older, garnered FDA priority review in March.

The agency expects to make a formal decision on maralixibat by late September, and it would be the first treatment for the rare liver disease.

“Bylvay and maralixibat work on a similar principle although using different mechanisms. The goal for each is to reduce the amount of bile acids returning to the liver, which reduces the amount of bile acid in the blood,” Vivian said. Trial results for both drugs were promising, with demonstrated improvements in quality of life and bile acid levels, according to Vivian.

“However, neither drug has been assessed for long-term liver benefit such as prevention of liver failure or reducing the need for liver transplantation. These long-term effects will be key to reducing the overall cost of care for these conditions,” Vivian said.