FDA approve first therapy to treat ultra-rare cancer

pharmafile | November 24, 2021 | News story | Research and Development  

Aadi Bioscience announced that the FDA have approved the intravenous drug Fyarro (sirolimus protein-bound particles for injectable suspension) for the treatment of locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumour, known as PEComa. Aadi shared that this approval marks the first therapy for this indication.

Malignant PEComas are a family of ultra-rare tumours that appear in fewer than one in a million people globally each year. PEComa is a group of soft tissue tumours that show perivascular epitheliod-cell (PEC) differentiation. They may be benign, malignant, or be of uncertain malignant potential. They are a form of sarcoma that form in soft tissues like the stomach, intestine, lungs and the female reproductive organs, along with genitourinary organs including the kidneys and bladder. Symptoms of malignant PEComa vary from one individual to another, and the presentations are based on the location and subtype of the tumour. PEComas may occur in different parts of the body.

The prognosis for patients with malignant PEComa is very poor, and after treatment with chemotherapy, median survival is around only 16 months. It is estimated that there are approximately 100 to 300 new patients per year in the US. Prior to the approval of Fyarro, there were no treatments specifically designed for PEComa patients. Nearly three quarters of patients will develop metastatic disease, most frequently to the lung or liver, within a year of diagnosis.

Neal Desai, founder, president and CEO of Aadi Bioscience, shared in a statement: “The approval of Fyarro is a momentous event not just for Aadi but, importantly, for advanced malignant PEComa patients. We reiterate that all of us at Aadi are incredibly grateful to all of the people with advanced malignant PEComa, their families and caregivers, as well as the healthcare professionals who made the Fyarro clinical studies possible.”

Ana Ovey

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