FDA Accepts Savara's MOLBREEVI Application for Rare Lung Disease with August Decision Date

Savara Inc. announced the FDA has accepted for review its resubmitted Biologics License Application for MOLBREEVI, a treatment for autoimmune pulmonary alveolar proteinosis. The FDA granted Priority Review with a target action date of August 22, 2026, under the Prescription Drug User Fee Act. Priority Review is reserved for applications that could represent significant improvements in safety or effectiveness for treating serious conditions.

MOLBREEVI is a recombinant human granulocyte-macrophage colony-stimulating factor delivered via nebulizer. The therapy aims to address autoimmune PAP, a rare lung disease where surfactant accumulates in the alveoli due to autoantibodies neutralizing GM-CSF, which prevents immune cells from clearing the substance properly. Management characterized autoimmune PAP as a rare lung disease with no approved pharmacological treatment option.

The CEO described Savara as a "single asset, rare disease company" focused on orphan rare pulmonary diseases. The company resubmitted its biologics license application at the end of December and expects to hear from the FDA "in the next couple of weeks" regarding acceptance and filing. The company's base case assumes MOLBREEVI will receive priority review due to its Breakthrough Therapy Designation, which would place a potential PDUFA decision "in the August timeframe."

Savara received an unexpected refusal to file in May of last year due to "in-process manufacturing data" the agency wanted before accepting the filing. Management believed the issue could have been resolved during the review, but the FDA required the additional information prior to filing acceptance. Following a Type A meeting with the FDA, Savara aligned with the agency on moving to an alternate drug substance manufacturer and on an analytical comparability protocol. The company completed a tech transfer to Fujifilm—an effort that had already been underway for about 18 months—and resubmitted the BLA in December with Fujifilm as the primary drug substance manufacturer. Management characterized the company as "very confident" in the resubmitted BLA.

The application includes data demonstrating the drug improves pulmonary gas transfer, quality of life, and clinical symptoms associated with the disease. The CEO described DLco as a surrogate endpoint and emphasized that, as a first mover in an orphan rare disease, Savara also viewed it as important to show clinical benefit in key secondary endpoints. The company highlighted robust results in the St. George's Respiratory Questionnaire measures and exercise tolerance testing using exercise treadmill tests. DLco reached statistical significance at 24 weeks (the primary endpoint) and remained statistically significant at 48 weeks as a key secondary endpoint, which the company said supported durability of effect.

The EVP of Global Medical Affairs outlined several reasons DLco was used and considered clinically meaningful in aPAP: it is well studied and used clinically to help diagnose autoimmune PAP, monitor treatment, and assess disease progression; it reflects the impact of surfactant burden on gas transfer, as it measures the diffusing capacity of the lungs for carbon monoxide; it is described as more responsive than other pulmonary function tests in aPAP patients treated with GM-CSF; and it is easy to perform and reproducible, with Savara standardizing equipment and training across trial sites.

On secondary endpoints, management referenced the concept of minimal clinically important difference for SGRQ established in other pulmonary diseases. SGRQ has not been validated specifically in autoimmune PAP but has been validated in conditions including COPD and lymphangioleiomyomatosis. A four-point reduction was cited as the MCID in COPD, and Savara observed that level of change in its SGRQ total and activity scores.

Savara plans to submit Marketing Authorization Applications to the European Medicines Agency and the UK's Medicines and Healthcare Products Regulatory Agency by the end of March 2026. The company has guided to filing marketing authorization applications in Europe and the U.K. by the end of the first quarter and stated that process remains on track.

MOLBREEVI has received multiple regulatory designations, including Fast Track, Breakthrough Therapy, and Orphan Drug Designation from the FDA. The European Medicines Agency has also granted Orphan Drug Designation, while the UK's MHRA awarded Innovation Passport and Promising Innovative Medicine designations. The name is conditionally accepted by both the FDA and EMA.

Autoimmune PAP causes shortness of breath, cough, and fatigue due to surfactant buildup in the lungs. The condition can lead to serious complications including lung fibrosis and potential need for lung transplant.

The Chief Commercial Officer said the company's claims database analysis identified approximately 5,500 diagnosed prevalent aPAP patients in the U.S. The company described "line of sight" as its process of profiling accounts associated with claims, confirming that patients are actively managed at those locations, and identifying who is currently managing them. Savara has reached a goal of having line of sight to 1,000 patients—about 20% of the market—through a small field effort, which the company said increased confidence that the market is "real and underappreciated." The company has narrowed pricing to $400,000–$500,000 per patient per year.

The company carries about $264M pro forma plus potential approval-linked non-dilutive funding, including a $75M RTW payment and up to $75–$150M from a restructured debt facility. The company amended its existing Loan and Security Agreement with Hercules Capital, enabling access to up to $105 million in term loans, contingent upon FDA approval of its MOLBREEVI product.

Savara is a clinical-stage biopharmaceutical company focused on rare respiratory diseases. MOLBREEVI represents the company's lead program and is not yet approved for any indication. The European Patent Office granted Savara and PARI a patent for their drug-device combination of MOLBREEVI and the eFlow Nebulizer System, offering protection through March 2043.