Savara Plans Rolling Submission to FDA for MOLBREEVI in Autoimmune Pulmonary Alveolar Proteinosis
核心洞察
Savara plans to initiate a rolling submission of the Biologics License Application (BLA) to the FDA for MOLBREEVI (搜索) by the end of the year.
The company anticipates completing the BLA submission by the end of Q1 2025 and intends to request priority review for potential U.S. approval by late 2025.
Savara expects to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) by the end of 2025.
Savara Inc. is advancing its regulatory strategy for MOLBREEVI (搜索), a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), as a potential treatment for autoimmune pulmonary alveolar proteinosis (搜索) (aPAP). Following a pre-BLA meeting with the FDA, the company plans to initiate a rolling submission of the Biologics License Application (BLA) by the end of 2024.
The company anticipates completing the BLA submission by the end of the first quarter of 2025 and intends to request priority review. If granted, this could lead to a potential approval in the U.S. by the end of 2025. In parallel, Savara is preparing to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) by the end of 2025.
Financial Position and Market Readiness
Savara reported a net loss of $24.2 million for the third quarter of 2024, compared to a net loss of $16.6 million for the same period in 2023. Research and development expenses increased to $20.3 million, driven primarily by activities related to the MOLBREEVI (搜索) program. As of September 30, 2024, the company held approximately $219.4 million in cash and short-term investments, which it believes is sufficient to fund operations through the second quarter of 2027.
"After a productive pre-BLA meeting with the FDA, we are working diligently to initiate a rolling submission for MOLBREEVI (搜索) by the end of this year, with plans to complete the BLA submission by the end of 1Q 2025—thus enabling a potential approval in the U.S. by the end of 2025, if priority review is granted," said Matt Pauls, Chair and CEO, Savara.
About Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Autoimmune PAP is a rare lung disease characterized by the accumulation of surfactant in the alveoli, impairing gas exchange and causing symptoms such as shortness of breath and fatigue. The condition arises when antibodies neutralize granulocyte-macrophage colony-stimulating factor (GM-CSF), hindering the ability of alveolar macrophages to clear surfactant effectively. Approximately 3,600 patients are diagnosed with aPAP in the U.S.
MOLBREEVI (搜索), delivered via an investigational eFlow Nebulizer System, aims to address this unmet need by providing recombinant human GM-CSF to stimulate surfactant clearance in aPAP patients.