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Orphan Drug Status Given to New Treatment for Mesothelioma

Published: September 1, 2023

A new treatment for mesothelioma was recently given orphan drug status by the United States FDA. The company given orphan drug status is known as Avenge Bio, which is a biotechnology company working to create a new immunotherapy treatment regimen. It announced that it was given orphan drug status back in June. People are being enrolled in phase 1 and phase 2 clinical trials for AVB-001. The cancers being tested include ovarian cancer, primary peritoneal mesothelioma, and fallopian tube cancer. Avenge Bio, the company that created the treatment, is developing AVB-001 using its LOCOcyte immunotherapy platform, which is used to treat solid tumors. This is the first time the treatment is being tested on humans. The researchers are looking to see how safe and tolerable the cancer treatment is in patients with certain types of cancer.

There are 44 patients being enrolled in the Avenge Bio study. The clinical trial is broken into two parts. The first phase is the dose escalation phase to find the highest possible dose and the second phase is a dose expansion phase. In the phase, there will be 20 extra platinum resistant, high grade, serous adenocarcinoma of the ovary, primary peritoneum, or fallopian tube patients. There could be other groups that could be tested in the second phase of the study to see if the treatment could be good as a singular therapy or as a combination with another treatment. The study is ongoing and there is an estimated completion date of August 2026.  To be eligible for the study, people must be 18 years or older, have a history of unresectable, platinum-resistant, high grade, serous carcinoma of the ovary, primary peritoneum, or fallopian tube, have an Eastern Cooperative Oncology Group performance status of 0 or 1 at screening, and not have had more than five lines of previous therapy. The clinical trial is being held at Massachusetts General Hospital in Boston, Women and Infants Hospital of Rhode Island in Providence, and MD Anderson Cancer Center in Houston.

Orphan Drug Status is given to certain drugs or treatments that are made for rare diseases that affect relatively small amounts of patients. Supporting development of drugs that work for rare diseases that only affect around 200,000 Americans creates benefits for drug makers. Benefits can include tax credits for qualified clinical trials, exemptions from user fees, and potential exclusive marketing for seven years after approval.

“Avenge Bio Receives FDA Orphan Drug Designation for AVB-001 for the Treatment of Mesothelioma” CISION PR Newswire (June 15, 2023). [Link]
“Avenge Bio secures FDA orphan drug designation for mesothelioma therapy” Pharmaceutical Technology (June 16, 2023).  [Link]
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