Savara's Molbreevi Receives FDA Priority Review with August 22 PDUFA Date for Autoimmune PAP

The U.S. Food and Drug Administration has accepted and filed Savara's Biologics License Application for Molbreevi (molgramostim inhalation solution) in autoimmune pulmonary alveolar proteinosis, granted priority review, and assigned a Prescription Drug User Fee Act action date of August 22, 2026. Savara also expects to file marketing authorization applications in the European Union and the United Kingdom by the end of March.

Molbreevi has the potential to be the first and only approved therapy for autoimmune PAP in the U.S. and Europe. There are currently no approved therapeutics for autoimmune PAP in the U.S., Europe, or the U.K. The disease is described as a serious, long-term, chronic, rare pulmonary condition with significant unmet need.

Data from the Phase III IMPALA program showed improvements in DLCO, SGRQ, exercise capacity and surfactant burden, supporting Molbreevi's mechanism of activating macrophages to address the underlying pathophysiology versus the procedure-based standard of care. Molbreevi is designed to address the pathophysiology of autoimmune PAP by activating macrophages, which in turn supports surfactant homeostasis and improves gas exchange. The therapy is intended to address key manifestations of autoimmune PAP, which include impaired pulmonary gas transfer, poor respiratory quality of life, reduced patient function, and surfactant burden.

Whole lung lavage is the primary existing intervention, characterized as a rescue procedure used in severe cases. The procedure is hospital-based, where patients are intubated and one lung is ventilated while the other lung is washed. The procedure is offered only in select centers, requires hospitalization and operating room and intensive care unit resources, and is not standardized, while also not addressing the underlying cause of disease.

Published epidemiology estimates vary widely, ranging from six to seven patients per million up to approximately 26 per million. Savara conducted claims database analysis and identified roughly 5,500 known and diagnosed patients in the U.S., which equates to about 16 patients per million and represents a "floor" for the U.S. market. The company believes all 5,500 patients could benefit from Molbreevi upon approval, citing feedback from physicians indicating they would offer the therapy regardless of disease severity.

The U.S. autoimmune PAP market appears concentrated, with the top 500 accounts managing roughly two-thirds of patients based on the company's claims analysis. Savara is planning a field presence of roughly 30 customer-facing personnel, including leadership, and expects to add a small number of field reimbursement specialists. The company is aiming to hire the field team in the second quarter to support disease awareness and testing education ahead of a potential approval.

Savara has chosen PANTHERx Rare as its exclusive specialty pharmacy partner in the U.S. The selection was based on PANTHERx's focus on rare and orphan diseases, experience with exclusive specialty pharmacy models, existing pulmonary relationships, and familiarity with device-based therapies. Savara is working with PANTHERx on pharmacy programming and patient services.

Savara reports $264 million cash plus up to $150 million in potential non-dilutive funding contingent on FDA approval. The company has a market capitalization of approximately $1.12 billion. Financial metrics show zero revenue over the trailing twelve months, reflecting its developmental stage status. The company's current ratio stands at 7.65, indicating robust short-term financial health, with a debt-to-equity ratio of 0.32, suggesting a conservative capital structure.