Savara Submits BLA for MOLBREEVI* to FDA for aPAP Treatment

Savara Inc., a biopharmaceutical company specializing in rare respiratory diseases, has announced the completion of its Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for MOLBREEVI. This submission is a pivotal step in seeking approval for MOLBREEVI as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP), a rare lung condition. According to Savara's Chair and CEO Matt Pauls, the data supporting the application illustrates that MOLBREEVI can significantly improve pulmonary gas exchange and alleviate symptoms associated with aPAP.

The company has requested Priority Review from the FDA, which, if granted, would expedite the review process from the usual ten months to six. This acceleration could facilitate an early 2026 commercial launch, pending approval. Savara's preparations for this potential launch are reportedly progressing well, reflecting its optimism about bringing MOLBREEVI to market.

The clinical efficacy of MOLBREEVI was demonstrated in the IMPALA-2 trial, which successfully met its primary endpoint. The trial showed that the treatment significantly enhanced the diffusing capacity of the lungs for carbon monoxide (DLCO), a critical measure of pulmonary gas exchange, compared to a placebo at the 24-week mark. Moreover, this improvement persisted through the 48th week, underscoring the treatment's durability.

Beyond gas exchange, MOLBREEVI also showed clinical benefits in other areas. Participants exhibited marked improvements in their scores on the St. George’s Respiratory Questionnaire (SGRQ), both in total score and activity level, as well as enhanced exercise capacity as measured by peak metabolic equivalents (peak METs). These findings, supported by significant statistical data, indicate that MOLBREEVI could provide comprehensive benefits for those with aPAP.

Safety profiles from the trial were favorable, with MOLBREEVI being well-tolerated by patients. A remarkable 97% of participants completed the trial's double-blind phase, and there were no dropouts due to drug-related adverse effects. This robust safety data will likely bolster the regulatory case for MOLBREEVI.

In addition to pursuing FDA approval, Savara is eyeing regulatory pathways in Europe. The company plans to submit a Marketing Authorization Application (MAA) by the end of 2025. MOLBREEVI has already received several designations that could facilitate its approval process, including Orphan Drug, Fast Track, and Breakthrough Therapy designations by the FDA and the European Medicines Agency. The UK’s Medicines and Healthcare Products Regulatory Agency has also granted it the Innovation Passport and Promising Innovative Medicine designations.

Autoimmune pulmonary alveolar proteinosis (aPAP) is characterized by the accumulation of surfactant in the lungs' alveoli, which impairs gas exchange and leads to symptoms like shortness of breath, cough, and fatigue. This condition arises because antibodies neutralize granulocyte-macrophage colony-stimulating factor (GM-CSF), inhibiting alveolar macrophages from clearing excess surfactant effectively. The chronic nature of aPAP can lead to serious complications like lung fibrosis and potential lung transplants.

Savara, through MOLBREEVI, aims to address these challenges, leveraging their experience in rare respiratory conditions and pulmonary medicine. MOLBREEVI, a recombinant form of GM-CSF, is delivered via a specialized nebulizer system, representing an innovative approach to aPAP treatment. The company's expertise and commitment are reflected in their efforts to bring this potentially groundbreaking therapy to patients who have limited treatment options.