The FDA removed the clinical hold after determining that UniQure had satisfactorily addressed all the issues the agency had identified. They related to a patient diagnosed with hepatocellular carcinoma in the Hope-B pivotal trial.
A surgical resection of the tumor and liver tissue from the patient who developed the disorder, done by an independent laboratory, showed that the patient's tissue sample was extremely rare and accounted for 0.03% of the cells in the tissue sample.
The trial was put on clinical hold in December after UniQure submitted a safety report relating to a possible related severe adverse event associated with the preliminary diagnosis of HCC in that patient.
It was later found that the patient may have been predisposed to this type of carcinoma.
“We do not anticipate any impact to our regulatory submission timeline for the hemophilia B program as a result of this clinical hold,” Chief Executive Matt Kapusta said in a statement at the time.
“Patient dosing is complete in each of uniQure’s three hemophilia B gene-therapy studies, and there is no plan to enroll or treat additional patients,” the company said.
All patients enrolled in the company's gene therapy program, including the 54 patients in the Hope-B trial, had had abdominal ultrasounds performed a year after dosing. Each will continue to be monitored.
UniQure shares at last check were 7.8% higher at $34.80.