FDA approves Fyarro for perivascular epithelioid cell tumor
Click Here to Manage Email Alerts
The FDA approved sirolimus protein-bound particles for injectable suspension (albumin-bound) for treatment of adults with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor.
Sirolimus protein-bound particles for injectable suspension (albumin-bound) [Fyarro, Aadi Bioscience] is a mammalian target of rapamycin (mTOR) inhibitor.
The agent is the first approved therapy for adults with malignant perivascular epithelioid cell tumor, a rare and aggressive form of sarcoma that occurs more frequently among women. Approximately 100 to 300 people are diagnosed in the United States each year, and estimated survival is 12 to 16 months.
Malignant perivascular epithelioid cell tumors often harbor mutations in the TSC1 or TSC2 genes that result in the activation of mTOR pathway.
The FDA based approval on results of the phase 2 AMPECT trial, which included 31 patients. Results per independent review showed an overall response rate of 39%. Two patients achieved complete response.
After median follow-up of 36 months, median duration of response had not been reached (range, 5.6-55.5+). Nearly all responses (92%) lasted at least 6 months; 67% lasted at least 12 months and 58% lasted at least 2 years.
Grade 3 nonhematologic events that occurred among more than 10% of patients included stomatitis, rash, fatigue and infections. Grade 3 laboratory abnormalities that occurred among more than 10% of patients included lymphocytopenia, increased glucose and decreased potassium.
“The approval of Fyarro ... will provide physicians with a new weapon for treating patients with this rare disease,” Andrew J. Wagner, MD, PhD, senior oncologist at Dana-Farber Cancer Institute and principal investigator in the AMPECT trial, said in an Aadi Bioscience-issued press release. “In our AMPECT trial, Fyarro demonstrated durable responses in mTOR inhibitor-naive patients with locally advanced unresectable or metastatic [perivascular epithelioid cell tumor], with an acceptable and manageable safety profile.”
The approval represents “a significant advance” for treating this disease, according to Robert G. Maki, MD, PhD, clinical director of the sarcoma program and professor of medicine at University of Pennsylvania.
“Patients living with locally advanced or metastatic [perivascular epithelioid cell tumor] are in urgent need of new treatment options,” Maki said in the release. “I am encouraged that Fyarro provided a clinically meaningful benefit in overall response rate, with some patients responding for up to several years. I am pleased to have Fyarro as a new therapeutic option to offer my [patients with advanced malignant perivascular epithelioid cell tumor].”
Read more about
Click Here to Manage Email Alerts