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UniQure Hemophilia Therapy Placed on FDA Clinical Hold

The FDA placed a uniQure program on clinical hold due to a safety issue: a possible link to liver cancer.
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Shares of uniQure  (QURE) - Get uniQure N.V. Report dropped on Monday after the Dutch-U.S. gene-therapy company said the U.S. Food and Drug Administration placed its hemophilia B gene-therapy program on clinical hold.

The FDA acted on a safety issue: a possible link to liver cancer. 

UniQure shares at last check traded at $37.93, down 17%. They have tumbled 47% year to date.

“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.

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“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.

“The clinical hold was initiated following the submission of a safety report in mid-December relating to a possibly related serious adverse event associated with a preliminary diagnosis of hepatocellular carcinoma, a form of liver cancer, in one patient in the Hope-B trial that was treated with etranacogene dezaparvovec (AMT-061) in October 2019.”

Further, “The patient has multiple risk factors associated with HCC, including a 25-year history of hepatitis C, hepatitis B virus, evidence of nonalcoholic fatty liver disease and advanced age,” the company said.

“We are working closely with the FDA and our advisers to conduct a thorough investigation into the cause of this event, which we expect to be completed in early 2021,” said Ricardo Dolmetsch, president of research and development at uniQure.

“We will investigate whether there is a relationship to treatment. At this time, we do not have adequate data to determine a possible causal relationship, especially in the context of the other known risk factors.”