LANGHORNE, PA — Savara Inc. (Nasdaq: SVRA) has announced the publication of a manuscript detailing long-term outcomes for molgramostim inhalation solution in patients with autoimmune pulmonary alveolar proteinosis (aPAP). The study, published in ERJ Open Research, highlights encouraging results that demonstrate the treatment’s ability to address the disease’s underlying pathophysiology and significantly improve patients’ quality of life.
The retrospective case series evaluated the outcomes of five aPAP patients treated with molgramostim through European single-patient access, with a mean treatment duration of 4.2 years. The data showed marked improvements in lung function, reduced disease burden, and enhanced ability to perform daily life activities. Pulmonary gas transfer, measured by percent predicted diffusing capacity of the lung for carbon monoxide (DLco) and alveolar-arterial oxygen gradient (A-aDO2), also demonstrated notable improvement.
Furthermore, high-resolution computed tomography scans revealed a reduction in surfactant burden in the lungs following treatment. Importantly, while four of the five patients required at least one whole lung lavage (WLL) prior to using molgramostim, no patients needed WLL after continuing treatment for more than one year. The therapy showed a strong safety profile, with no reported serious adverse events, and was well tolerated.
“Long-term outcomes data from these case studies are encouraging and support our belief that treatment with molgramostim may address the root cause of aPAP, resulting in improved and sustained patient benefit,” said Matt Pauls, Chair and Chief Executive Officer at Savara.
Molgramostim works by stimulating alveolar macrophages that are otherwise impaired by antibodies neutralizing granulocyte-macrophage colony-stimulating factor (GM-CSF), a critical factor in clearing excess surfactant that builds up in aPAP patients. This uncontrollable buildup causes impaired gas exchange, resulting in severe symptoms and potential long-term complications such as lung fibrosis or reliance on a lung transplant.
Savara’s recent findings bolster the company’s commitment to advancing molgramostim as the first pharmaceutical treatment option for aPAP in the U.S. and Europe. The company remains on track to complete its rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration by the end of the first quarter of 2025, with plans to submit a Marketing Authorization Application (MAA) in Europe by year-end.
With these advancements, Savara aims to provide relief and improved quality of life for aPAP patients, who currently have limited treatment options.
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