FDA Approves Shionogi Liver Disease Med

Shionogi & Co., Ltd., based in Osaka, Japan and Florham Park, New Jersey, announced that the U.S. Food and Drug Administration (FDA) approved Mulpleta (lusutrombopag) for thrombocytopenia in adults with chronic liver disease who were about to have a medical procedure performed.

Mulpleta is a once-daily, oral medication that acts as a small molecule agonist on the thrombopoietin (TPO) receptor. It is part of a new class of drugs known as thrombopoietin receptor agonists (TPO-RA). They stimulate platelet production. Thrombocytopenia is often seen as a complication of CLD, occurring in up to 78 percent of patients with cirrhosis. Patients with CLD and thrombocytopenia are at increased risk of bleeding and require recurrent platelet transfusions, more ambulatory visits and inpatient hospital stays.

“Therapeutic options in this area are critically needed, as adult patients with chronic liver disease often require multiple procedures for various medical reasons,” said Nezam Afdhal,senior physician in Hepatology at Beth Israel Deaconess Medical Center and professor of Medicine at Harvard Medical School, in a statement. “I look forward to being able to offer this new oral treatment to patients, instead of relying solely on platelet transfusions.”

The FDA approval was based on safety and efficacy results from two Phase III clinical trials, L-PLUS 1 and L-PLUS 2.

In May, Durham, North Carolina-based Dova Pharmaceuticals announced the FDA had approved its Doptelet (avatrombopag) for thrombocytopenia in adults with CLD. It was the first TPO-RA approved in the U.S. for that indication.

“We are delighted FDA has approved Doptelet, which represents a significant milestone for Dova, physicians, and most importantly, patients,” said Alex Sapir, president and chief executive officer of Dova, in a statement in May. “Doptelet is the first orally administered treatment option for patients with CLD, allowing the majority of patients to avoid a platelet transfusion prior to a procedure by increasing platelet counts to the target level of greater or equal to 50,000 per microliter. Given our extensive preparations to date, we are positioned to launch Doptelet in June with our full complement of sales, marketing, and reimbursement support resources.”

Shionogi’s Mulpleta has been used in Japan since it was approved in September 2015 by the Ministry of Health, Labor and Welfare. The FDA had a PDUFA date of August 26 for the drug, but zipped it through approval almost a month early. In two studies, 78 percent and 65 percent of patients receiving the drug did not require transfusions ahead of medical procedures. The patients also didn’t require rescue therapy for bleeding within 7 days of the procedure.

“Patients with chronic liver disease and thrombocytopenia undergoing non-emergent invasive procedures currently have no approved medical treatment options except for platelet transfusions, therefore therapeutic options in this area are critically needed,” said Tsutae “Den” Nagata, Shionogi’s chief medical officer, in a statement last fall.

The drug has been validated by the European Medicines Agency (EMA) and is expected to be approved in the first half of 2019. It is expected to be available in the U.S. by early September 2018.

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