According to Savara, the FDA has granted Breakthrough Therapy designation for Molgradex inhaled recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP).
In June 2019, Savara announced that the Phase 3 IMPALA study of Molgradex for that indication had failed to meet its primary endpoint. In October, the company said that it had held a Type C meeting with the FDA and that the agency’s written response recommended against submission of a biologics license application (BLA) submission for Molgradex at that time.
Savara CEO Rob Neville said, “We are pleased that the FDA recognized Molgradex as a breakthrough therapy for the treatment of aPAP, a debilitating rare lung disease with no approved pharmacologic treatment options. We believe this designation reflects the significance of Molgradex as an investigational product that, based on the IMPALA study, has been demonstrated to improve patient outcomes. Breakthrough designation is designed to provide increased collaboration and more frequent dialogue with the FDA and is an important milestone as we work to determine the best path forward for this product.”
The company is also developing Molgradex for the treatment of nontuberculous mycobacterial (NTM) lung infections in cystic fibrosis patients and in non-CF patients.
Read the Savara press release.
