Savara Inc. has established an expanded access program for molgramostim nebulizer solution to assist adult patients diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP). According to information from ClinicalTrials.gov, the program provides access to the investigational drug for individuals with this rare disorder.
Autoimmune pulmonary alveolar proteinosis is a rare condition in which surfactant builds up in the lungs, making breathing difficult. Symptoms can include shortness of breath, persistent cough, fatigue, weight changes, and chest or back pain. While whole lung lavage is a common invasive treatment, it carries risks such as lung trauma or the need for artificial ventilation.
Molgramostim nebulizer solution is an inhaled investigational drug designed to activate cells that help clear surfactant from the lungs to improve oxygen transfer. In clinical trials, the solution has shown improvements in patient reported outcomes and gas exchange. Under this expanded access program, eligible adult patients receive a 300 mcg dose once daily via inhalation, with physician follow-ups every three months to monitor clinical status and adverse events.
The program is available to adults who meet specific criteria, including a positive serum anti-granulocyte macrophage colony-stimulating factor (GM CSF) autoantibody test and a history of aPAP confirmed through biopsy, cytology, or imaging. Access to the treatment must be obtained through a treating physician.
