FDA Accepts Savara's MOLBREEVI Application for Autoimmune PAP with Priority Review

Savara Inc. (Nasdaq: SVRA), a clinical-stage biopharmaceutical company focused on rare respiratory diseases, announced the FDA has filed for review the BLA for MOLBREEVI as a therapy to treat patients with autoimmune PAP. The FDA granted Priority Review with a PDUFA action date of August 22, 2026.

The company plans to submit the MOLBREEVI Marketing Authorization Applications (MAAs) for Autoimmune PAP to the European Medicines Agency (EMA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) by the end of Q1 2026. MOLBREEVI has the potential to be the first and only approved therapy for autoimmune PAP in the U.S. and Europe.

FDA Priority Review designation directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard review applications.

In addition to Fast Track and Breakthrough Therapy Designations, MOLBREEVI has been granted Orphan Drug Designation for the treatment of autoimmune PAP by the FDA and the EMA, as well as Innovation Passport (IP) and Promising Innovative Medicine (PIM) designations by the UK's MHRA.

MOLBREEVI is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis. MOLBREEVI is delivered via an investigational eFlow® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of a large molecule.

Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli. Surfactant consists of proteins and lipids and is an important physiological substance that lines the alveoli to prevent them from collapsing. In a healthy lung, excess surfactant is cleared and digested by immune cells called alveolar macrophages. Alveolar macrophages need to be stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function properly in clearing surfactant, but in autoimmune PAP, GM-CSF is neutralized by autoantibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas transfer, resulting in clinical symptoms of shortness of breath, often with cough and frequent fatigue. Patients may also experience episodes of fever, chest pain, or coughing up blood, especially if secondary lung infection develops. In the long term, the disease can lead to serious complications, including lung fibrosis and the need for a lung transplant.

MOLBREEVI is the FDA and EMA conditionally accepted trade name for molgramostim inhalation solution. It is not approved in any indication.