FDA hustles through a quick OK for Shionogi's new thrombocytopenia drug
The FDA has whisked through a new drug for thrombocytopenia patients.
The therapy is lusutrombopag (Mulpleta), which handily beat the August 26 PDUFA date that the FDA had handed Shionogi when it accepted the drug for a priority review schedule back in February.
The drug is approved for use in thrombocytopenia patients — characterized by low platelet levels — with chronic liver disease who are about to undergo an invasive, scheduled medical or dental procedure. The drug proved itself in two studies in which a large majority of the patients — 78% and 65% — required no transfusions ahead of procedures after getting the drug, compared to much smaller groups in the placebo arms.
To be counted a success, patients also didn’t need any rescue therapy for bleeding within 7 days of their procedure. As it stood at the finish line, bleeding as an adverse event occurred in the drug arm of one pivotal group at half the rate of the 5.6% seen in the placebo arm.
“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, last fall.
The drug, once dubbed S-888711, is an oral small molecule agonist of the human thrombopoietin receptor. It’s been used in Japan for close to three years.
This is one drug that barely registered on the industry radar as it passed through late-stage tests and on into the FDA. The agency didn’t even feel it worth worth a standard endorsement in its approval today.
It ranks as the 26th new drug approval for the year.