Savara said that the FDA provided a written response after a Type C meeting regarding the company’s planned biologics license application (BLA) submission for Molgradex inhaled recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) in which the agency recommended against submission of the BLA at this time due to insufficient efficacy and safety data. Savara said that it is “working to determine the next steps for the Molgradex development program.”
In 2017, Savara said that it had met with the FDA for guidance on how to modify the Phase 3 IMPALA study of Molgradex for aPAP to qualify as a pivotal study and that changes included increasing enrollment. In June 2019, the company announced that the study had failed to meet its primary endpoint.
Savara CEO Rob Neville said, “While we are disappointed in the FDA’s response, considering the IMPALA study results presented today at the ERS annual conference, we remain committed to the Molgradex development program and believe that it will provide aPAP patients with a meaningful treatment option. Our priority is to further assess the content of the FDA’s feedback and determine the best development path forward.”
Savara is also developing Molgradex for the treatment of nontuberculous mycobacterial (NTM) lung infection in cystic fibrosis patients and announced the initiation of a Phase 2a trial for that indication in April 2019.
Read the Savara press release.
