Savara Pharmaceuticals
Savara Pharmaceuticals focuses on rare respiratory diseases and is developing molgramostim, an inhaled GM-CSF, for autoimmune pulmonary alveolar proteinosis (aPAP), currently in Phase 3 trials.
Services
Savara Pharmaceuticals focuses on developing therapies for rare respiratory diseases. Their lead product candidate, molgramostim, is designed to treat autoimmune pulmonary alveolar proteinosis (aPAP). The company also provides expanded access policies for patients who need molgramostim but are outside of clinical trials.
Products
The primary product candidate from Savara Pharmaceuticals is molgramostim, an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF). It is currently in Phase 3 development for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP). Molgramostim aims to activate macrophages in the lung alveoli to restore surfactant-clearing activity and improve oxygenation in aPAP patients.
Regulatory Designations
Molgramostim, Savara Pharmaceuticals' lead product candidate, has been granted several key designations by various regulatory bodies. These include Orphan Drug Designation from both the U.S. FDA and the European Medicines Agency (EMA), Fast Track and Breakthrough Therapy Designations by the U.S. FDA, and the Innovation Passport (IP) and Promising Innovative Medicine (PIM) designations by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA).
Clinical Trials
Savara Pharmaceuticals has conducted the IMPALA trial, the largest controlled clinical trial for autoimmune pulmonary alveolar proteinosis (aPAP). The trial was conducted in 18 countries to evaluate the effectiveness of molgramostim. The current standard-of-care for aPAP is an invasive procedure called Whole Lung Lavage (WLL), and molgramostim aims to offer a less invasive alternative.
Headquarters
Savara Pharmaceuticals is headquartered in Langhorne, Pennsylvania. The company also maintains a Clinical Advisory Board that focuses on autoimmune pulmonary alveolar proteinosis (aPAP), helping guide clinical development and research efforts.