Savara Resubmits the Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for MOLBREEVI* for the Potential Treatment of Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune PAP)
Savara Resubmits the Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for MOLBREEVI* for the Potential Treatment of Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune PAP)
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biologics license applicationregulatory
A biologics license application is a formal request submitted to regulatory authorities seeking approval to market a new biological medicine, such as vaccines or treatments made from living organisms. It is a comprehensive review process that evaluates the safety, effectiveness, and manufacturing quality of the product. For investors, receiving approval signals that a biological therapy can be sold to the public, potentially leading to revenue growth and market success.
fdaregulatory
The FDA is the U.S. federal agency that evaluates and approves medical drugs, devices, biological therapies and certain foods; think of it as the gatekeeper that decides whether a medical product is safe and effective for patients. For investors, FDA decisions determine whether a company can sell a product, affect expected revenue and introduce regulatory risk, so approvals, rejections or safety warnings can quickly move a company's valuation and stock price.
autoimmune pulmonary alveolar proteinosismedical
A rare lung disease in which the tiny air sacs that exchange oxygen become filled with excess protein and fat, like a sponge clogged with foam so it can’t hold air properly. It matters to investors because it creates specific needs for diagnostics, treatments and long-term care; the rarity and complexity drive regulatory scrutiny, clinical trial risk and potential niche market opportunities for companies developing drugs or medical devices.
surfactantmedical
A surfactant is a chemical that helps liquids spread, mix, or stick by lowering the tension at the boundary between them—think of soap helping oil and water mingle or wetting a surface like water on a glass. For investors, surfactants matter because they are essential ingredients in many products and medicines, affect manufacturing quality and costs, and can influence regulatory approval, product performance, and supply‑chain risks for companies that make or rely on them.
alveolimedical
Alveoli are tiny air sacs at the ends of the lung’s airways where oxygen moves into the blood and carbon dioxide is removed, like millions of tiny balloons exchanging gases with nearby blood vessels. Their health and function matter to investors because many drugs, vaccines, medical devices and regulatory decisions hinge on how well treatments reach or protect these sacs; damage or improvement in alveoli can change clinical trial results, patient outcomes and market demand for respiratory products.
fast trackregulatory
A fast track designation is a regulatory label that speeds up the review and communication between a drug developer and regulators for treatments addressing serious illnesses or unmet medical needs. For investors, it matters because it can shorten development time and reduce regulatory delays—like getting a VIP lane at the airport—raising the chance of earlier market access and potential revenue, though it does not guarantee approval.
breakthrough therapy designationsregulatory
A breakthrough therapy designation is a special status granted by a regulatory agency to a drug or treatment that shows strong early signs of being substantially better than current options for a serious illness; it gives the developer faster and more intensive guidance during testing and review. For investors, the designation can shorten time to market, lower development risk and costs, and increase the odds of a valuable approval — like giving a promising product a fast-pass through the approval process.
orphan drug designationregulatory
Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.
-- The BLA was Resubmitted with FUJIFILM Biotechnologies (Fujifilm) as the Drug Substance Manufacturer and Priority Review was Requested --
LANGHORNE, Pa.--(BUSINESS WIRE)--
Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, announced today that it has resubmitted the MOLBREEVI BLA to the FDA, with Fujifilm as the drug substance manufacturer. The MOLBREEVI BLA submission is seeking approval for the treatment of autoimmune PAP, a chronic and debilitating rare lung disease characterized by the abnormal build-up of surfactant in the alveoli. The Company has requested Priority Review of the application.
MOLBREEVI was granted Fast Track and Breakthrough Therapy Designations by the FDA, Orphan Drug Designation by the FDA and by the European Medicines Agency (EMA), and Innovation Passport (IP) and Promising Innovative Medicine (PIM) designations by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of autoimmune PAP.
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 antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas exchange, 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.
Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, 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. Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at www.savarapharma.com and LinkedIn.
*MOLBREEVI is the FDA and EMA conditionally accepted trade name for molgramostim inhalation solution. It is not approved in any indication. MOLBREEVI is a trademark of Savara Inc.
