StemCell Therapy

Even the most promising drugs can hit a pothole in the road to U.S. Food and Drug Administration (FDA) approval, particularly when working in the complicated, evolving world of gene therapies.

After touting positive Phase III resultsat ASH (American Society of Hematologys annual meeting),uniQuregot slapped with anFDA clinical holdfor their hemophilia B treatment due to a patient developing liver cancer.

Theresearch team alerted the FDA of the development after a lesion was found during a routine one-yearfollow-up.A hepatocellular carcinoma (HCC) isexpectedto be confirmed with a full surgical resection.

However, the cancer may not be related to the gene therapy, as this particular patient was high risk for liver cancerwith a long history of hepatitis C, hepatitis B virus, findings of non-alcoholic fatty liver disease and advanced age.UniQurepointed out thatchronicinfections with hep B and C are associated with 80% of HCC cases.

Patient safety will always be our top priority, and we are working closely with the FDA and our advisors to conduct a thorough investigation into the cause of this event which we expect to be completed in early 2021, said RicardoDolmetsch, Ph.D., president of R&D atuniQure. 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.

The hold is not expected to affect the pivotal trial results as the dosing of the patients is completed.

While several treatments areavailable for hemophilia B, doctors and patients are holding out hope for a cure that will stop the disease at its source. Thats thepotential that genetic medicine has held for this area.

UniQuresdrug, AMT-061, substantially increased production of the blood-clotting protein factor IX, thefactor missing forhemophiliaBpatients,innearly all trial participants with moderate to severe disease.

Drug developers have run into a challenge with gene therapies in patients with high levels of neutralizing antibodies. Those antibodies can disarma gene therapyby attacking its viral vector delivery systembefore it ever has the chance to help thepatient.Many gene therapy trials wont enroll patients who test positive for neutralizing antibodies.

UniQuresgene therapy is delivered by an AAV5 vehicle, whichis believed to be harder to impair than other vectors.The company enrolled 23 patients with neutralizing antibodiespresent.Only one patient did not respondto the therapy. That was one who had a large amount of theantibodies- encouraging results that at least some with neutralizing antibodies could go on the therapy.

All patients in our hemophilia B gene therapy program, including the 54 patients in HOPE-B, will continue to be monitored by their care teams while we gather additional information as rapidly as possible, noted CEO Matt Kapusta. We do not anticipate any impact to our regulatory submission timeline for the hemophilia B program as a result of this clinical hold.

Featured Jobs on BioSpace

Read more here:
Hope on Hold: Promising Hemophilia B Therapy Paused by FDA - BioSpace

This entry was posted on December 26, 2020 at 1:57 pm and is filed under Genetic medicine. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |