Savara reported that the U.S. FDA granted Fast Track designation for Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF).
Molgradex is currently being investigated in a pivotal Phase 3 study, IMPALA, for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP). Topline results from the study are expected in June 2019. Positive results would facilitate the submission of a Biologics License Application (BLA) in the first half of 2020, with an anticipated commercial launch later in 2020 or early 2021.
The Fast Track program facilitates the expedited development and review of new drugs or biologics that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. A drug granted Fast Track designation may be eligible for Priority Review or Rolling Review of the BLA, if relevant criteria are met.
About autoimmune pulmonary alveolar proteinosis
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare autoimmune lung disorder. It is the most common form (90% of the cases) of pulmonary alveolar proteinosis (PAP). Most cases affect adults between the ages of 20-50 years. Some people may not show symptoms, while others may have progressive difficulty breathing and shortness of breath upon exertion. Other signs and symptoms may include a dry, chronic cough; fatigue; weight loss; chest pain; and a general feeling of ill health. Autoimmune PAP is caused by an immune system malfunction, due to IgG antibodies that block the granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) effect. GM-CSF is a protein that regulates clearance of surfactant (a mix of protein and fat) by alveolar macrophages. The surfactant pile up in the air sacs of the lungs (alveoli), and eventually lead to an inability to breath.
Molgradex is an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF). Molgradex was granted Orphan Drug Designation for the treatment of PAP in the United States and the European Union.