Discovering a treatment for a rare disease is challenging but it can also be rewarding. This is especially true when treating an extremely rare disease such as perivascular epithelioid cell tumor (PEComa). PEComa occurs on an average of one case per million individuals. About one hundred to three hundred people are diagnosed with PEComa each year in the U.S.
Biospace published an article highlighting the announcement by Aadi Bioscience that the FDA approved its drug Fyarro for PEComa. Fyarro is an mTOR inhibitor used in the treatment of renal cancer and was recently studied for other types of cancers such as advanced PEComa.
Aadi Bioscience has since announced the U.S. Commercial Launch of FYARRO™ for the treatment of adult patients with advanced metastatic malignant PEComa that cannot be surgically removed (unresectable).
This year Aadi will present multiple posters at the American Association for Cancer Research Annual Meeting 2023 to be held April 14-19 2023 in Orlando, Florida. One poster is entitled “Biomarker analysis from AMPECT correlating response to nab-sirolimus with TSC1 and TSC2 inactivating alterations“.
About PEComas
Perivascular epithelioid cell tumor (PEComa) is defined as mesenchymal tumors that are composed of perivascular epithelioid cells. They are rare soft tumors that can form around small blood vessels in body parts, for example, the GI tract, lungs, kidneys, uterus, or liver.
PEComa prognosis can be very poor with the patient’s life extended about sixteen months after chemotherapy treatment. Within a year after diagnosis, three-fourths of patients will experience metastatic disease usually in the liver or lung.
The majority of PEComas tend to be benign. However, some may be malignant and spread to other body parts. The cell type of origin is unknown.
About Aadi Bioscience
The company, Aadi Bioscience, is focused on therapies that apply to genetically defined cancers. Aadi brings therapies to patients with alterations to the mTOR pathway.
Looking Back
The name perivascular epithelioid cell tumor was first proposed in the early 1990s after researchers noticed shared cellular features between the various tumors that are now members of the PEComa grouping. These features are considered unique to these types of tumors, distinguishing them from other tumors.
Aadi stated that the FDA approval is a first for PEComa. Fyarro had been approved under a priority review and was made available commercially in February 2022.
Aadi’s CEO, Brendan Delaney, speculated that Fyarro is slated to become the standard of care for cases of advanced malignant PEComa.
