Savara Launches EAP for Molgramostim for aPAP Patients

LANGHORNE, Pa.--(BUSINESS WIRE)--Savara Inc. (Nasdaq: SVRA), a clinical stage biopharmaceutical company focused on rare respiratory diseases, has unveiled the Savara Early Access Program. This initiative is an Expanded Access Program (EAP) for molgramostim, targeting patients with autoimmune Pulmonary Alveolar Proteinosis (aPAP). The program allows physicians to request molgramostim for eligible aPAP patients in regions where the drug is not yet commercially available, adhering to local regulatory guidelines.

The U.S. Food and Drug Administration (FDA) has reviewed and permitted the Savara Early Access Program to proceed. Currently, the program is open to eligible patients in selected countries across North America and Europe, with expansion plans set through 2026.

"Expanded access is granted to investigational products that address a serious condition where no similar therapies exist," explained Matt Pauls, Chair and CEO of Savara. "Given the significant unmet need in aPAP and the positive results from the Phase 3 IMPALA-2 clinical trial, we deemed it crucial to establish this program. Our aim is to offer eligible aPAP patients pre-approval access to molgramostim, reflecting our commitment to the global aPAP community and our objective of delivering an effective therapy for this rare lung disease as swiftly as possible."

Savara plans to submit a Biologics License Application for molgramostim to the FDA in the first half of 2025. Molgramostim has received several designations from the FDA, including Orphan Drug, Fast Track, and Breakthrough Therapy. It has also been granted Orphan Drug designation by the European Medicines Agency and both Innovative Passport and Promising Innovative Medicine designation by the UK's Medicines and Healthcare Products Regulatory Agency for the treatment of aPAP.

### About Expanded Access to Molgramostim

Expanded Access Programs (EAPs) serve as a potential pathway for patients with serious or life-threatening diseases to access investigational medical treatments outside of clinical trials before commercial availability, especially when no comparable or satisfactory alternative therapies exist. Healthcare professionals and aPAP patients interested in Savara's EAP for molgramostim can learn more by visiting clinical trials websites or contacting Savara directly.

### About aPAP

Autoimmune Pulmonary Alveolar Proteinosis (aPAP) is a rare lung disease marked by the abnormal accumulation of surfactant in the alveoli, the tiny air sacs in the lungs. Surfactant is a crucial substance composed of proteins and lipids that lines the alveoli to prevent collapse. In healthy lungs, excess surfactant is cleared by alveolar macrophages, immune cells that need stimulation by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function correctly. However, in aPAP, antibodies neutralize GM-CSF, impairing macrophages' ability to clear surfactant, leading to surfactant buildup in the alveoli. This results in impaired gas exchange, manifesting as shortness of breath, cough, and fatigue. Patients may also experience fever, chest pain, or hemoptysis, especially if secondary lung infections occur. Over time, aPAP can lead to severe complications, including lung fibrosis and the need for a lung transplant.

### About Savara

Savara Inc. is a clinical stage biopharmaceutical firm specializing in rare respiratory diseases. Their primary program involves molgramostim inhalation solution, a recombinant human GM-CSF in Phase 3 development for aPAP. Molgramostim is administered via an investigational eFlow® Nebulizer System, designed specifically for the inhalation of large molecules. Savara's management team has extensive experience in rare respiratory diseases and pulmonary medicine, focusing on unmet needs and advancing product candidates toward approval and commercialization. For more information, visit Savara's official website.