rare respiratory diseases

Savara Inc. announced positive Phase 3 trial results for molgramostim in autoimmune pulmonary alveolar proteinosis (aPAP) at ATS 2025. In 157 patients, the therapy significantly reduced radiological surfactant burden (mean GGO score decrease 2.1 vs. 1.1; P=0.0004) and need for rescue whole-lung lavage (7.4% vs. 13.3%). Molgramostim improved respiratory quality of life metrics (St. George’s Respiratory Questionnaire, P=0.0084) and reduced breathing difficulties (P=0.0305 and P=0.0049 at Weeks 24/48). By addressing impaired GM-CSF signaling, this inhaled therapy could redefine aPAP treatment, offering a non-invasive alternative to current procedures. Trial data supports future regulatory submissions for rare disease approval pathways.