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FDA grants priority review to Shionogi's lusutrombopag

Published 27 February 2018

Shionogi's new drug application (NDA) for lusutrombopag (S-888711) has been accepted for filing and granted priority review by the US Food & Drug Administration (FDA).

Lusutrombopag (S-888711) is an investigational, once-daily, orally administered, small molecule thrombopoietin (TPO) receptor agonist.

In the US, Shionogi is seeking FDA approval of lusutrombopag for the treatment of thrombocytopenia in patients with chronic liver disease who are at increased risk for bleeding associated with invasive procedures.

The submission is based on two Phase 3 clinical trials, L-PLUS1 and L-PLUS2, in which lusutrombopag met the pre-specified primary and all key secondary endpoints with statistically significant results. The Prescription Drug User Fee Act (PDUFA) date for an FDA decision is August 26, 2018.

Shionogi president and CEO John Keller said: “I am very pleased the FDA has granted Priority Review for lusutrombopag’s New Drug Application. This step emphasizes an urgent need exists for more advanced medicines for the treatment of thrombocytopenia in patients living with chronic liver disease (CLD) who have to undergo invasive procedures.”

 “We at Shionogi look forward to the upcoming FDA review, and the near future in which patients and physicians have additional, advanced therapeutic options beyond platelet transfusions which are the current standard of care.”

The FDA Priority Review status accelerates the review time from a standard 10-month review to a goal of six (6) months from the date of acceptance of filing.

A Priority Review designation will direct overall attention and resources to the evaluation of applications that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.

The European Medicines Agency has validated for review Shionogi’s standard Marketing Authorization Application (MAA) for lusutrombopag. In Europe, the MAA submission is based on the same two Phase 3 clinical trials as the FDA filing.

Lusutrombopag (S-888711) is an orally administered, small molecule agonist of the human thrombopoietin receptor. Lusutrombopag was approved by the Ministry of Health, Labor and Welfare in Japan in September 2015 for the improvement of thrombocytopenia associated with CLD in patients undergoing an elective invasive procedure.

Thrombocytopenia is a common complication of CLD, which may be caused by multiple factors including decreased production of TPO. Thrombocytopenia is frequently observed in patients with CLD, with studies suggesting that it occurs in up to 78% of patients with cirrhosis.1

CLD-associated thrombocytopenia is defined as a platelet count of less than 150,000/µL and is the most common hematologic complication of CLD.

Patients with CLD and thrombocytopenia are at increased risk for bleeding, requiring recurrent platelet transfusions, increased ambulatory visits and inpatient hospital stays compared with patients with CLD without thrombocytopenia.5

The annual health care cost of a patient with CLD with thrombocytopenia is more than three times that of a patient with CLD without thrombocytopenia.5 In addition to the potential of thrombocytopenia, especially severe thrombocytopenia (platelet count less than 50,000/µL), to aggravate surgical or traumatic bleeding, it may also significantly complicate routine diagnostic procedures and patient care, such as liver biopsy and medically indicated or elective procedures for cirrhotic patients, resulting in delayed or cancelled curative treatment.6

Source: Company Press Release