Savara Reports 50% Increase in Autoimmune PAP Patient Population, Plans December 2025 FDA Resubmission for MOLBREEVI
Key Insights
Savara's updated health claims analysis identifies approximately 5,500 autoimmune PAP (search) patients in the U.S., representing a 50% increase from the company's previous 2023 estimate of 3,600 patients.
The revised analysis used a more comprehensive dataset with over three times the number of records compared to the previous study, suggesting improved awareness and diagnosis rates for this rare lung disease.
The company plans to resubmit its Biologics License Application for MOLBREEVI (search) to the FDA in December 2025 with priority review request, following publication of positive Phase 3 IMPALA-2 trial results in the New England Journal of Medicine.
Savara Inc. (Nasdaq: SVRA) announced that a comprehensive analysis of U.S. health claims data has identified approximately 5,500 patients currently diagnosed with autoimmune pulmonary alveolar proteinosis (search) (autoimmune PAP (search)), representing a nearly 50% increase over the company's previous 2023 estimate of about 3,600 patients. The clinical-stage biopharmaceutical company, focused on rare respiratory diseases, commissioned the updated analysis as it advances toward resubmitting its Biologics License Application (BLA) for MOLBREEVI (search) in December 2025.
Enhanced Data Analysis Reveals Larger Patient Population
The revised analysis was conducted by a third-party advanced analytics provider using a more recent and comprehensive dataset that accessed more than three times the number of records compared to the 2023 study. PAP patients were identified using ICD-10 diagnostic code J84.01 and required a medical or pharmacy claim within the last two and a half years.
The analysis applied additional validation criteria to confirm autoimmune PAP (search) cases, including the number of PAP diagnoses, visits to specialty physicians such as pulmonologists, relevant procedures, and documented symptoms related to autoimmune PAP. Since autoimmune PAP accounts for over 90% of all PAP cases, prevalence rates were applied to the confirmed PAP population to arrive at the estimated 5,500 autoimmune PAP patients in the U.S.
"The increase in U.S. autoimmune PAP (search) prevalence, based on recent data from a large and robust data source, may indicate that awareness and diagnosis rates are accelerating," said Braden Parker, M.B.A., Chief Commercial Officer at Savara. "As a data-driven organization with deep expertise and experience in rare disease commercialization, we are committed to capturing the most accurate insights to increase awareness of this rare and debilitating lung disease and fuel earlier detection."
MOLBREEVI Advances Toward FDA Resubmission
Savara is finalizing the analytical data package for its BLA resubmission to the FDA, scheduled for December 2025, with plans to request priority review. The company recently announced that results from its pivotal Phase 3 IMPALA-2 clinical trial were published in the New England Journal of Medicine, demonstrating that MOLBREEVI (search) addresses the underlying pathophysiology of autoimmune PAP (search).
"With no approved medicines in the U.S. to treat autoimmune PAP (search), these claims data reinforce our belief that there is a large unmet medical need as we push toward resubmitting the MOLBREEVI (search) BLA," said Matt Pauls, M.B.A., J.D., Chair and Chief Executive Officer of Savara. "Strong operational execution over the last quarter has put the Company on a solid path forward."
MOLBREEVI (search) has received multiple regulatory designations recognizing its potential therapeutic value, including Fast Track and Breakthrough Therapy Designations from the FDA, Orphan Drug Designation from both the FDA and the European Medicines Agency (search) (EMA), as well as Innovation Passport (IP) and Promising Innovative Medicine (PIM) designations from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA).
Understanding Autoimmune PAP
Autoimmune PAP (search) is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli of the lungs. In healthy lungs, surfactant balance is maintained by immune cells called alveolar macrophages, which require stimulation by granulocyte-macrophage colony-stimulating factor (search) (GM-CSF) to function properly in clearing surfactant.
In autoimmune PAP (search), GM-CSF is neutralized by antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. This results in excess surfactant accumulation in the alveoli, causing impaired gas exchange and clinical symptoms including shortness of breath, cough, and frequent fatigue. Patients may also experience episodes of fever, chest pain, or coughing up blood, particularly if secondary lung infection develops. Long-term complications can include lung fibrosis (search) and the potential need for lung transplantation.
MOLBREEVI (search) is a recombinant human granulocyte-macrophage colony-stimulating factor (search) (GM-CSF) delivered via a proprietary investigational eFlow® Nebulizer System specifically developed for inhalation of the therapy. The treatment is designed to restore the function of alveolar macrophages in clearing excess surfactant from the lungs.