FDA Grants Priority Review to Hairy Cell Leukemia Treatment

The United States Food and drug Administration recently announced its decision to grant a priority review. The privilege of expedited process, according to a press release, falls to AstraZeneca and MedImmune. The companies developed a drug known as moxetumomab pasudotox for the treatment of hairy cell leukemia. Keep reading to learn more or follow the original story for additional details.

AstraZeneca and MedImmune designed Moxetumomab pasudotox as a drug specialized for the treatment of hairy cell leukemia in adults. Specifically, researchers created the drug for patients having received at least two previous forms of therapy. The drug has also previously gone by the names/codes CAT08015 and HA22.

Functioning as an anti-CD22 recombinant immunotoxin, investigation drug moxetumomab pasudotox could be first-in-class treatment for its target population.

In simplest terms, the drug functions by binding to a specific protein on the cell membrane (CD22) which allows it to enter the cancer cells and destroy them.

An ongoing phase 3 “1053” trial tests the treatments efficacy, and safety, among other qualities, among a population of 80 patients with relapse or refractory hairy cell leukemia.

Researchers administered doses of moxetumomab pasudotox to patients participating in the trial on every first, third, and fifth day of a 28-day cycle. Patients could participate in up to six cycles. Researchers watched for disease progression, dangerous levels of toxicity, or a complete response. The start of an alternate therapy would also cause the cycles to be halted.

This single-arm trial, staged across multiple centers, met its primary endpoint of a durable and complete response. Researchers intend to present their results at a soon to be announced medical meeting according to a press release. Estimates pace completion of the study in May of 2018.

Approval of this new drug could mean hope for a lot of hairy cell leukemia patients. The rare blood disorder affects around 1000 people in the United States each year and there is currently no standard of care treatment protocol. Many patients respond well to initial rounds of therapy and treatment. Just under half of them, however, suffer from a relapse some time later.

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