LANGHORNE, PA — Savara Inc. (Nasdaq: SVRA) has announced receiving a Refuse-to-File (RTF) letter from the U.S. Food and Drug Administration (FDA) for its Biologics License Application (BLA) of MOLBREEVI, a potential treatment for autoimmune pulmonary alveolar proteinosis (autoimmune PAP). The letter, issued after a preliminary review, noted that the BLA submitted in March 2025 was incomplete, requiring additional Chemistry, Manufacturing, and Controls (CMC) data.
Crucially, the FDA’s RTF was not due to safety concerns, and no further efficacy studies were requested. Savara plans to request a Type A meeting with the FDA within the next 30 days to discuss the data requirements. “The requested CMC data outlined in the RTF letter are currently being generated, and we look forward to meeting with the FDA to align on next steps,” said Matt Pauls, Chair and CEO of Savara. He added that the company expects to resubmit the BLA in the fourth quarter of 2025.
MOLBREEVI has already received several regulatory designations, including Fast Track and Breakthrough Therapy by the FDA, Orphan Drug status in both the U.S. and Europe, and innovation-focused labels from the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA). These designations underscore the potential significance of MOLBREEVI in addressing the rare and debilitating disease.
As part of its manufacturing strategy, Savara is advancing efforts to establish a redundant supply chain. Pauls stated, “We remain on track to complete the technology transfer with our second-source drug substance contract manufacturer in the fall,” highlighting progress on various production qualification batches and analytical comparability efforts.
Autoimmune PAP is a rare lung disease marked by the abnormal buildup of surfactant in the alveoli, impairing gas exchange and leading to symptoms like shortness of breath, fatigue, and, in some cases, severe complications. MOLBREEVI aims to address this condition by improving pulmonary function and patients’ respiratory health-related quality of life.
Savara remains confident in its ability to meet the FDA’s requirements and in the potential of MOLBREEVI to improve the lives of individuals affected by this rare disease.
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