Patients facing serious illnesses who have exhausted all available treatment options often want to know if and how they can receive access to investigational drugs that haven’t yet been approved by the FDA. The information below explains options that are available to patients in these circumstances, specifically with regard to aTyr’s investigational product, efzofitimod.
For new medicines to be legally approved for use, companies like aTyr are required to evaluate their safety and effectiveness in clinical trials and submit trial results to the FDA. To participate in a trial, you must meet certain criteria. For those who meet the criteria to join a clinical trial, participation offers the chance to contribute to medical research that may benefit many others. Participation in a clinical trial comes with certain risks; that is why patient “informed consent” is a required step in the process of enrolling.
Efzofitimod is being investigated as a treatment for pulmonary sarcoidosis in a Phase 3 trial, EFZO-FIT™.
Clinical Expanded Access or Compassionate Use
Expanded access, also referred to as compassionate use, is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Whenever possible, an investigational medical product should be used as part of a clinical trial. However, if patient enrollment is not possible (e.g., patient ineligibility, lack of ongoing clinical trials) or enrollment in a clinical trial is not feasible (e.g., distance to a trial precludes access), expanded access offers a possible route for gaining access to an investigational product.
In order to focus on development efforts to gain regulatory approval for use in patients, aTyr does not currently intend to provide efzofitimod to patients through an expanded access program.