FDA Fast-Tracks Drug for Rare Autoimmune Lung Disorder

Inhaled Form of Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)

Orphan lung disease company Savara Inc. has received an FDA fast-track designation for Molgradex, an inhaled form of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF). The drug is being investigated in a pivotal phase 3 study, IMPALA, for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP).

Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare autoimmune lung disorder. Most cases involve adults aged between 20 and 50 years. Some people show no symptoms, but others can have difficulty breathing and shortness of breath upon exertion; dry, chronic cough; fatigue; weight loss; chest pain; and a general feeling of ill health.

Autoimmune aPAP is caused by an immune system malfunction, a result of IgG antibodies blocking the GM-CSF effect. The protein GM-CSF regulates clearance of surfactant by alveolar macrophages. The surfactant builds up in the alveoli then eventually leads to the inability to breathe. The standard treatment is lung lavage.

Topline results from IMPALA are expected in June 2019. Positive results will lead to Savara submitting a Biologics License Application in the first half of 2020, and the company anticipates a commercial launch later that year or in early 2021.

Molgradex, a GM-CSF in phase 3 development for aPAP, is also in phase 2a development for nontuberculous mycobacterial (NTM) lung infection in patients with non-cystic fibrosis (CF) and patients who have CF with chronic NTM lung infection.

Source: Savara Inc., May 6, 2019