Discovering a treatment for a rare disease is challenging and rewarding, especially when it is an extremely rare disease such as perivascular epithelioid cell tumor (PEComa).
Biospace recently carried an article highlighting an announcement by Aadi Bioscience that the FDA approved its drug Fyarro for PEComa. This is the first approved therapy for PEComa.
About PEComas
Malignant PEComas usually occur in soft tissues such as lungs, stomach, female reproductive or genitourinary organs, and intestines.
PEComas are found in one person out of approximately one million individuals. There are about one hundred to three hundred new cases in the U.S. every year. The disease affects women disproportionately.
PEComas are extremely aggressive with a poor prognosis. Treating the disease with chemotherapy will only extend a patient’s life between 12 to 16 months.
Within a year after diagnosis, the PEComas will have spread (metastasized) primarily in the liver or lung in about 75% of patients.
About the Phase II Study
Data from the Phase II trial resulted in a response rate of 39% after treatment with Fyarro. Two patients achieved complete responses.
Although a large majority of patients experienced a response that lasted over twelve months, the median duration of thirty-six months was not reached.
About Fyarro
Treatment with Fyarro, an mTOR inhibitor, achieved impressive responses in patients who had either metastatic (spreading) or inoperable (unresectable) PEComa. The protein mTOR is associated with the control of several cell functions. It is known that blocking mTOR can kill cancer cells.
The company added that prescribing information for Fyarro runs along the same lines as its warnings related to stomatitis, myelosuppression, infections. Boxed warnings can be found here.
Looking Forward
Fyarro is expected to be available in the first quarter of 2022. The drug was approved for priority review which expedites the approval process.
Aadi’s COO, Brendan Delaney, said that in view of Fyarro’s clinical success, the medication is expected to be a standard of care in cases of malignant PEComa.
