Expanded Access to nab-sirolimus
About Expanded Access:
In cases where a clinical trial isn’t an option, and the patient has exhausted all available treatment options, investigational drug may be provided before it is commercially available. Such individual use of an investigational drug pre-approval is often called “expanded access” or “compassionate use”.
For a patient to obtain access to an investigational drug through expanded access, the patient’s physician, the drug’s sponsor, and in certain countries, the regional regulatory authority must all approve the use.
Expanded Access Program for Advanced Malignant PEComa
Aadi Bioscience, Inc. (Aadi) is committed to helping patients with cancer obtain access to new treatments. Aadi manufactures FYARRO® (nab-sirolimus; ABI-009) which has been approved in the United States for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa), i.e. “advanced malignant PEComa”. Outside of the United States, safety and efficacy has not been established for nab-sirolimus in advanced malignant PEComa, and any such use is investigational.
While we believe access to our investigational medicines is best accomplished through participation in clinical trials, we recognize that this is not always possible for every cancer patient. In certain situations, Aadi may be able to provide nab-sirolimus through our Expanded Access Program outside of the United States.
This page describes our policy on providing expanded access to nab-sirolimus.
Requests for expanded access will be evaluated on a case-by-case basis and will also be contingent upon whether Aadi has adequate supply of investigational product and whether there is a regulatory mechanism in the country or region to support expanded access.
To begin the process, a physician must submit a request to Aadi (Contact: firstname.lastname@example.org) and acknowledge that the patient for whom the request is being submitted meets the criteria below.
1. The patient has received a diagnosis of advanced malignant PEComa and:
- Patient is ≥18 years of age
- Diagnosis of advanced malignant PEComa is confirmed by pathology and immunohistochemistry
2. The potential benefits to the patient outweigh the potential risks
3. The provision of nab-sirolimus will not interfere with the ongoing development program
We strive to respond to the physician within 3 business days once a request for expanded access is submitted for a patient. Additional information may be required before a determination can be made.
It’s important to remember that investigational drugs have not yet received regulatory approval; therefore, their potential benefits and potential risks are not yet established. Patient preferences, along with clinical judgment of the treating healthcare professional, should be used to determine the optimal treatment approach for each individual patient.