— Company Expects to Complete BLA Submission by End of 1Q 2025 —
LANGHORNE, PA, USA I December 18, 2024 I Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, initiated a rolling submission of a BLA to the FDA for MOLBREEVI for the potential treatment of aPAP, a chronic and debilitating rare lung disease characterized by the abnormal build-up of surfactant in the alveoli of the lungs.
MOLBREEVI was granted Fast Track and Breakthrough Therapy Designations in 2019 for the treatment of patients with aPAP. As a result, the Company is allowed to submit individual modules of the BLA as they are completed rather than waiting to submit the application once all modules are available. The Company will request a priority review of the BLA when the submission is completed.
“Given the positive results of the pivotal, Phase 3 IMPALA-2 trial, we believe MOLBREEVI demonstrates a favorable benefit-risk profile and could fundamentally change the way aPAP is treated,” said Matt Pauls, Chair and Chief Executive Officer, Savara. “Initiation of the BLA is an important milestone in potentially addressing the unmet need in aPAP, for which there are no approved medicines in the U.S. and Europe. We look forward to working closely with the FDA throughout the review process and expect to complete the submission of the rolling BLA by the end of 1Q 2025.”
In addition to Fast Track and Breakthrough Therapy Designations, MOLBREEVI has been granted Orphan Drug Designation for the treatment of aPAP by the FDA and by the European Medicines Agency (EMA), Innovation Passport (IP) and Promising Innovative Medicine (PIM) designations by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA).
About Autoimmune PAP
Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli (or air sacs) of the lungs. 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 aPAP, 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.
About Savara
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 (aPAP). MOLBREEVI is delivered via an investigational eFlow® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of a large molecule. 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, X: @SavaraPharma and LinkedIn.
*MOLBREEVI is the FDA and EMA conditionally accepted trade name for molgramostim inhalation solution.
SOURCE: Savara
Post Views: 2,652
— Company Expects to Complete BLA Submission by End of 1Q 2025 —
LANGHORNE, PA, USA I December 18, 2024 I Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, initiated a rolling submission of a BLA to the FDA for MOLBREEVI for the potential treatment of aPAP, a chronic and debilitating rare lung disease characterized by the abnormal build-up of surfactant in the alveoli of the lungs.
MOLBREEVI was granted Fast Track and Breakthrough Therapy Designations in 2019 for the treatment of patients with aPAP. As a result, the Company is allowed to submit individual modules of the BLA as they are completed rather than waiting to submit the application once all modules are available. The Company will request a priority review of the BLA when the submission is completed.
“Given the positive results of the pivotal, Phase 3 IMPALA-2 trial, we believe MOLBREEVI demonstrates a favorable benefit-risk profile and could fundamentally change the way aPAP is treated,” said Matt Pauls, Chair and Chief Executive Officer, Savara. “Initiation of the BLA is an important milestone in potentially addressing the unmet need in aPAP, for which there are no approved medicines in the U.S. and Europe. We look forward to working closely with the FDA throughout the review process and expect to complete the submission of the rolling BLA by the end of 1Q 2025.”
In addition to Fast Track and Breakthrough Therapy Designations, MOLBREEVI has been granted Orphan Drug Designation for the treatment of aPAP by the FDA and by the European Medicines Agency (EMA), Innovation Passport (IP) and Promising Innovative Medicine (PIM) designations by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA).
About Autoimmune PAP
Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli (or air sacs) of the lungs. 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 aPAP, 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.
About Savara
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 (aPAP). MOLBREEVI is delivered via an investigational eFlow® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of a large molecule. 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, X: @SavaraPharma and LinkedIn.
*MOLBREEVI is the FDA and EMA conditionally accepted trade name for molgramostim inhalation solution.
SOURCE: Savara
Post Views: 2,652
