Savara Inc. announced that the MHRA has accepted the submission of the MOLBREEVI MAA for the treatment of autoimmune PAP in the U.K. The MAA was accepted under Accelerated Review and qualifies for a 150-day assessment duration. A decision on the application is expected in Fourth Quarter 2026. In the U.S., the FDA is reviewing the MOLBREEVI BLA under Priority Review with an August 22, 2026 Prescription Drug User Fee Act (PDUFA) date.
In Europe, the MOLBREEVI MAA is being reviewed by the Committee for Medicinal Products for Human Use (CHMP) with a decision expected in First Quarter 2027. In addition to Fast Track and Breakthrough Therapy Designations in the U.S., MOLBREEVI has been granted Orphan Drug Designation for the treatment of autoimmune PAP by the FDA and the EMA, as well as Innovation Passport and Promising Innovative Medicine designations by the MHRA. 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 a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (autoimmune PAP). MOLBREEVI is delivered via a proprietary investigational eFlow Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of MOLBREEVI.
