Savara Begins BLA Submission to FDA for MOLBREEVI to Treat aPAP

Savara Inc., a biopharmaceutical company based in Langhorne, Pennsylvania, is making strides in the treatment of rare respiratory diseases. The company has begun a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for their drug candidate, MOLBREEVI, aimed at treating autoimmune pulmonary alveolar proteinosis (aPAP). This chronic lung disease is marked by an abnormal accumulation of surfactant in the alveoli, the tiny air sacs in the lungs.

MOLBREEVI has received significant attention and regulatory support due to its potential benefits. The drug was granted Fast Track and Breakthrough Therapy Designations back in 2019, allowing Savara to submit parts of their BLA as they are completed, rather than waiting for a complete application. This regulatory pathway is designed to expedite the development and review of drugs that offer substantial improvements over existing therapies. Once the application is fully submitted, the company plans to seek priority review, which could further accelerate the approval process.

Savara’s CEO, Matt Pauls, expressed optimism about the prospects for MOLBREEVI, citing favorable outcomes from the Phase 3 IMPALA-2 trial. He highlighted the importance of the BLA submission milestone in potentially providing a new treatment option for aPAP, a condition for which there are currently no approved medications in the United States or Europe. Savara aims to complete the BLA submission by the end of the first quarter of 2025 and intends to maintain close collaboration with the FDA during the review period.

Beyond the U.S., MOLBREEVI has also received Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA), indicating its potential to address a significant unmet medical need in a rare disease. Additionally, the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted it the Innovation Passport and Promising Innovative Medicine designations, further underscoring its potential impact.

Autoimmune PAP is a rare disorder caused by the build-up of surfactant, a substance composed of proteins and lipids that normally prevents alveoli from collapsing. In healthy individuals, alveolar macrophages, stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF), clear excess surfactant. However, in aPAP, antibodies against GM-CSF hinder these macrophages from performing their function, leading to surfactant accumulation. This causes symptoms such as shortness of breath, coughing, fatigue, and sometimes fever or chest pain. If untreated, aPAP can result in severe complications, including lung fibrosis or the need for a transplant.

Savara’s lead program, MOLBREEVI, which is in Phase 3 development, is a recombinant human GM-CSF delivered through an investigational eFlow Nebulizer System. This device is specifically engineered for the inhalation of large molecules. The company’s management team brings extensive expertise in addressing rare respiratory conditions and is focused on advancing promising therapies towards regulatory approval and market introduction.

In summary, Savara Inc., through its innovative approaches and strategic regulatory engagements, is working towards introducing MOLBREEVI as a viable treatment option for patients suffering from aPAP. The company's ongoing efforts may offer new hope to those affected by this challenging and rare respiratory disease.