On 22 November 2021, the US Food and Drug Administration (FDA) approved sirolimus protein-bound particles for injectable suspension (albumin-bound) (Fyarro, Aadi Bioscience, Inc.) for adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumour (PEComa).<\/p>\n
Efficacy was evaluated in AMPECT (NCT02494570), a multicentre, single-arm clinical study in 31 patients with locally advanced unresectable or metastatic malignant PEComa. Patients received sirolimus protein-bound particles at 100 mg\/m2<\/sup> on days 1 and 8 of each 21-day cycle until disease progression or unacceptable toxicity.<\/p>\n The main efficacy outcome measures were overall response rate (ORR) and duration of response (DoR), as assessed by blinded independent central review, using RECIST v1.1. The ORR was 39% (95% confidence interval [CI] 22%, 58%), including 2 patients with complete responses. Median DoR was not reached (95% CI 6.5 months, not estimable). Among responders, 67% had a response lasting greater than 12 months and 58% had a response lasting greater than 24 months.<\/p>\n The most common (\u226530%) adverse reactions were stomatitis, fatigue, rash, infection, nausea, oedema, diarrhoea, musculoskeletal pain, decreased weight, decreased appetite, cough, vomiting, and dysgeusia. The most common (\u22656%) grade 3 to 4 laboratory abnormalities were decreased lymphocytes, increased glucose, decreased potassium, decreased phosphate, decreased haemoglobin, and increased lipase.<\/p>\n The recommended dosage is 100 mg\/m2<\/sup> administered as an i.v. infusion over 30 minutes on days 1 and 8 of each 21-day cycle until disease progression or unacceptable toxicity.<\/p>\n Full prescribing information for Fyarro is available here<\/a>.<\/p>\n This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA\u2019s assessment.<\/p>\n This application was granted priority review, fast track designation, breakthrough therapy designation, and orphan drug designation.<\/p>\n Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA\u2019s MedWatch Reporting System.<\/p>\n For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact FDA\u2019s Oncology Center of Excellence<\/a> Project Facilitate<\/a>.<\/p>\n