Promising liver disease drugs in pipeline for 2021-2022



According to PBM OptumRx, three notable drugs to treat liver disease are expected to go live in Q3 2021 and 2022.

According to PBM OptumRx, three notable drugs to treat liver disease are expected to go live in Q3 2021 and 2022.

A key drug to watch for 2022 is Intercept Pharmaceuticals’ obeticholic acid for non-alcoholic steatohepatitis (NASH), said Savitha Vivian, senior vice president of clinical services and forms at OptumRx. Managed Health Care Leader®.

NASH is a very common chronic liver disease without FDA-approved treatment, Vivian noted. Obeticholic acid was initially rejected by the FDA in 2020 when the agency asked Intercept for more data, and the agency’s recent full response letter “determined that OCA’s predicted benefit based on a Alternative histopathologic endpoint remains unclear and does not sufficiently outweigh the potential risks to support the expedited approval of treatment for patients with hepatic fibrosis due to NASH, ”Intercept said in a press release.

The FDA recommends that Intercept submit additional post-interim analytical data on the efficacy and safety of its ongoing REGENERATE study in support of possible expedited approval.

“We may get more data and a possible resubmission to the FDA 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,

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

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

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

“PFIC does not generally respond to conventional treatments used off-label, such as ursodeoxycholic acid, antihistamines or rifampicin. The only proven effective treatments are surgery and transplantation, ”she said.

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

Meanwhile, Miriam Pharmaceuticals’ maralixibat, a sodium-dependent apical bile acid transporter (ASBT) inhibitor taken orally to treat cholestatic pruritus in patients with Alagille syndrome (ALGS) aged one and over. plus, got priority review from the FDA in March.

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

“Bylvay and maralixibat work on a similar principle although they use different mechanisms. Everyone’s goal is to reduce the amount of bile acids going back into the liver, which reduces the amount of bile acids 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 evaluated for long-term hepatic benefit, such as preventing liver failure or reducing the need for liver transplantation. These long-term effects will be critical in reducing the overall cost. care for these conditions, ”said Vivian.


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