FDA seeks additional data for Savara’s MOLBREEVI BLA

LANGHORNE, Pa. - Savara Inc. (NASDAQ:SVRA), a biopharmaceutical company specializing in rare respiratory diseases with a market capitalization of $598 million, recently encountered a regulatory hurdle as the U.S. Food and Drug Administration (FDA) issued a Refuse to File (RTF) letter for the company’s Biologics License Application (BLA) concerning MOLBREEVI, a proposed treatment for autoimmune pulmonary alveolar proteinosis (PAP). The news triggered a 9.4% decline in the stock price over the past week, though InvestingPro analysis suggests the company remains slightly undervalued at current levels.

The FDA’s preliminary review, conducted after the BLA submission in March 2025, concluded that the application lacked completeness, specifically in the Chemistry, Manufacturing, and Controls (CMC) data section. The agency has not raised safety concerns nor has it requested additional efficacy studies. Savara plans to seek a Type A meeting with the FDA within the next 30 days to discuss the RTF and align on the requirements for a resubmission of their BLA, which is anticipated to occur in the fourth quarter of 2025. According to InvestingPro data, the company maintains a strong liquidity position with a current ratio of 14.91, suggesting ample resources to navigate this regulatory process. For deeper insights into Savara’s financial health and 10 additional ProTips, consider exploring InvestingPro’s comprehensive analysis tools.

Matt Pauls, Chair and Chief Executive Officer of Savara, expressed confidence in the company’s ability to address the FDA’s requests, citing ongoing efforts to generate the requested CMC data. He also emphasized Savara’s commitment to establishing a redundant supply chain, including the completion of a technology transfer with a second-source drug substance contract manufacturer scheduled for fall.

The RTF letter does not affect MOLBREEVI’s previously granted regulatory designations, which include Fast Track and Breakthrough Therapy Designations by the FDA, Orphan Drug Designation by the FDA and the European Medicines Agency (EMA), and Innovation Passport (IP) and Promising Innovative Medicine (PIM) designations by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA).

Autoimmune PAP is a rare condition marked by an accumulation of surfactant in the lungs, leading to impaired gas exchange and respiratory symptoms. MOLBREEVI, which is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) delivered via a nebulizer, aims to improve pulmonary function and quality of life for patients with this disease.

The information in this article is based on a press release statement from Savara Inc. and does not include any analysis or commentary on the reported facts.

In other recent news, Savara Inc. has completed its Biologics License Application (BLA) for Molbreevi, a treatment for autoimmune pulmonary alveolar proteinosis (aPAP). JMP Securities and H.C. Wainwright have both maintained positive outlooks on Savara, with price targets of $10 and $6, respectively. JMP Securities is optimistic about the FDA accepting the BLA by May, potentially granting a Priority Review due to the urgent need for new aPAP treatments. Analysts at JMP have assigned a 90% probability of success for Molbreevi’s approval, citing its Breakthrough Therapy designation and compelling Phase 3 trial results.

Additionally, Savara is preparing to present data from its Phase 3 clinical trial at upcoming medical congresses in Germany and Japan, focusing on the effectiveness of molgramostim in improving patient outcomes. In a strategic move, Savara has terminated its sales agreement with Evercore Group L.L.C., originally allowing for the sale of up to $100 million in shares. The termination will take effect in April 2025, with no penalties incurred. Savara’s recent developments, including the launch of its aPAP ClearPath blood test, aim to enhance diagnosis and market readiness for Molbreevi, with projected initial annual sales between $300 million and $500 million.

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