In the human body, TSC1 and TSC2 proteins inhibit mammalian target of rapamycin (mTOR) in the form of a complex. Studies have shown that in malignant perivascular epithelioid cell tumors, there is activation of the mTOR pathway due to TSC1 or TSC2 gene mutations, leading to abnormal proliferation of tumor cells. Therefore, mTOR is a potential therapeutic target for malignant perivascular epithelioid cell tumors.
The survival time of malignant perivascular epithelioid cell tumors exceeds 3 years
The mechanism of action of mTOR inhibitor-Fyarro(sirolimus) albumin
And Fyarro(sirolimus) albumin (Fyarro), developed by Aadi Biopharmaceuticals, is an mTOR inhibitor. Fyarro(sirolimus) albumin can bind to the FK506-binding protein-12 (FKBP-12) receptor in cells to produce an immunosuppressive complex that binds to and inhibits the activation of the rapamycin complex 1 (mTORC1) mechanism target, thereby reducing tumor cell proliferation, angiogenesis and glucose uptake, and exerting a therapeutic effect.
Fyarro(sirolimus) albumin, the first treatment for rare sarcomas, is approved for marketing
On November 22, 2021, the U.S. Food and Drug Administration (FDA) approved Fyarro(sirolimus) albumin for marketing, which is suitable for adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid tumors. This means that Fyarro(sirolimus) albumin has become the first and only drug approved by the FDA for the treatment of advanced malignant perivascular epithelioid tumors in adults.
Malignant perivascular epithelioid tumors achieve durable remission, with a survival time of 40.8 months
In the AMPECT trial, researchers explored the clinical efficacy and safety of Fyarro(sirolimus) albumin for patients with locally advanced unresectable or metastatic malignant perivascular epithelioid tumors. The primary endpoints of the trial were objective response rate (ORR) and duration of response (DOR).
1. Efficacy results
The objective response rate of patients treated with Fyarro(sirolimus) albumin was 39%, of which 2 patients achieved complete remission (CR), and the median duration of remission has not yet been reached. The median progression-free survival (PFS) of patients was 10.6 months, and the median overall survival (OS) was 40.8 months. Fyarro(sirolimus) albumin greatly prolonged the survival time of patients. Among the patients who experienced remission, 67% of the patients had a remission duration of more than one year, and 58% of the patients had a remission duration of more than two years.
2. Safety results
In terms of safety, most adverse reactions associated with Fyarro(sirolimus) albumin treatment were grade 1-2, and these adverse reactions were controllable through long-term treatment. No adverse reactions related to treatment of grade ≥ 4 occurred during treatment.
The trial confirmed that Fyarro(sirolimus) albumin is active in patients with malignant perivascular epithelioid cell tumors, can achieve durable remission and has good safety.
How much does Fyarro(sirolimus) albumin cost per box?
As early as after Fyarro(sirolimus) albumin was launched, Brendan Delaney, former chief operating officer of Aadi, pointed out that Fyarro(sirolimus) albumin is expected to become a standard treatment for advanced malignant perivascular epithelioid cell tumors. In 2022, Fyarro(sirolimus) Albumin was recognized by the NCCN Guidelines and became the only preferred treatment option for malignant perivascular epithelioid cell tumors.
[Warm Tips]: The above prices are only reference prices, and the actual prices may fluctuate due to exchange rates and other factors.
