Savara Begins BLA Submission to FDA for MOLBREEVI for aPAP Treatment

LANGHORNE, Pa.—Savara Inc., a biopharmaceutical company that focuses on rare respiratory conditions, recently commenced a progressive submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for MOLBREEVI. This investigational therapy is designed to treat autoimmune pulmonary alveolar proteinosis (aPAP), a chronic, debilitating disorder marked by surfactant accumulation in the lung alveoli.

MOLBREEVI received Fast Track and Breakthrough Therapy Designations in 2019, which facilitates the submission process by allowing the company to file parts of the BLA as they are completed. This strategy is expected to expedite the overall review, and Savara plans to seek priority review once the BLA is fully submitted.

Matt Pauls, the Chair and CEO of Savara, expressed optimism about MOLBREEVI's impact. "Following the promising results from the Phase 3 IMPALA-2 trial, we are confident that MOLBREEVI offers a strong benefit-risk ratio and has the potential to revolutionize aPAP treatment," he stated. Pauls emphasized the significance of beginning the BLA submission, given there are no approved aPAP treatments in both the U.S. and Europe. Savara anticipates completing the BLA submission by the end of the first quarter of 2025.

In addition to its Fast Track and Breakthrough Therapy Designations, MOLBREEVI has earned Orphan Drug Designation for aPAP from both the FDA and the European Medicines Agency (EMA). The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has also granted it Innovation Passport and Promising Innovative Medicine designations.

Autoimmune PAP is a rare pulmonary condition caused by excessive surfactant build-up in the alveoli. Surfactant, composed of proteins and lipids, is essential for preventing alveolar collapse. In a healthy system, alveolar macrophages clear surplus surfactant effectively. However, in aPAP, antibodies neutralize granulocyte-macrophage colony-stimulating factor (GM-CSF), impairing macrophages' ability to clear surfactant. Consequently, the buildup hinders gas exchange, leading to symptoms like breathlessness, cough, and fatigue. Patients may also suffer from fever, chest pain, or hemoptysis, especially with secondary infections. Long-term, the disease can result in serious complications such as lung fibrosis, potentially necessitating a lung transplant.

Savara is committed to addressing this unmet medical need. Its primary development program, MOLBREEVI, is a recombinant human GM-CSF, currently in Phase 3 trials for aPAP treatment. Delivered via an investigational eFlow® Nebulizer System designed for large molecules, the therapy is aimed at significantly improving patient outcomes. Savara's leadership team brings extensive expertise in rare respiratory diseases and is well-versed in bringing new therapies from development to market.

Through its innovative approach, Savara is poised to make substantial advancements in aPAP treatment, and the ongoing BLA submission marks a crucial step in potentially providing a new therapeutic option for patients with this challenging condition.