Savara Initiates Rolling Submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for MOLBREEVI* for the Potential Treatment of Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Savara Inc. (Nasdaq: SVRA) has initiated a rolling submission of a Biologics License Application (BLA) to the FDA for MOLBREEVI, targeting the treatment of autoimmune Pulmonary Alveolar Proteinosis (aPAP), a rare lung disease. The company expects to complete the BLA submission by the end of Q1 2025 and will request priority review.
MOLBREEVI has received multiple regulatory designations including Fast Track, Breakthrough Therapy, and Orphan Drug Designation from both FDA and EMA. The drug demonstrated positive results in the Phase 3 IMPALA-2 trial, showing a favorable benefit-risk profile. If approved, MOLBREEVI would be the first approved treatment for aPAP in the U.S. and Europe.
Savara Inc. (Nasdaq: SVRA) ha avviato una presentazione progressiva di una Domanda di Licenza Biologica (BLA) alla FDA per MOLBREEVI, mirata al trattamento dell'autoimmune Pulmonary Alveolar Proteinosis (aPAP), una malattia polmonare rara. L'azienda prevede di completare la presentazione della BLA entro la fine del primo trimestre del 2025 e richiederà una revisione prioritaria.
MOLBREEVI ha ricevuto numerose designazioni regolatorie tra cui Fast Track, Breakthrough Therapy e Orphan Drug Designation sia dalla FDA che dall'EMA. Il farmaco ha mostrato risultati positivi nella fase 3 dello studio IMPALA-2, evidenziando un profilo di beneficio-rischio favorevole. Se approvato, MOLBREEVI sarebbe il primo trattamento approvato per aPAP negli Stati Uniti e in Europa.
Savara Inc. (Nasdaq: SVRA) ha iniciado una presentación progresiva de una Solicitud de Licencia Biológica (BLA) ante la FDA para MOLBREEVI, dirigido al tratamiento de la Proteinosis Alveolar Pulmonar autoinmune (aPAP), una enfermedad pulmonar rara. La compañía espera completar la presentación de la BLA para finales del primer trimestre de 2025 y solicitará una revisión prioritaria.
MOLBREEVI ha recibido múltiples designaciones regulatorias que incluyen Fast Track, Breakthrough Therapy y Orphan Drug Designation tanto de la FDA como de la EMA. El fármaco mostró resultados positivos en el ensayo de fase 3 IMPALA-2, evidenciando un perfil de beneficio-riesgo favorable. Si se aprueba, MOLBREEVI sería el primer tratamiento aprobado para aPAP en los EE. UU. y Europa.
사바라 Inc. (Nasdaq: SVRA)는 자가면역 폐포 단백질증 (aPAP)이라는 희귀 폐 질환 치료를 위해 FDA에 MOLBREEVI에 대한 생물학적 라이센스 신청(BLA)의 롤링 제출을 시작했습니다. 회사는 2025년 1분기 말까지 BLA 제출을 완료할 것으로 예상하며, 우선 심사를 요청할 예정입니다.
MOLBREEVI는 FDA와 EMA 모두에서 패스트 트랙, 획기적인 요법, 희귀의약품 지정 등 여러 규제 지정을 받았습니다. 이 약물은 3상 IMPALA-2 시험에서 긍정적인 결과를 보여주며 유리한 이익-위험 프로필을 입증했습니다. 승인이 되면 MOLBREEVI는 미국과 유럽에서 aPAP에 대한 첫 번째 승인 치료제가 될 것입니다.
Savara Inc. (Nasdaq: SVRA) a lancé une soumission progressive d'une Demande de Licence Biologique (BLA) à la FDA pour MOLBREEVI, visant à traiter la protéinose alvéolaire pulmonaire auto-immune (aPAP), une maladie pulmonaire rare. L'entreprise prévoit de compléter la soumission de la BLA d'ici la fin du premier trimestre 2025 et demandera un examen prioritaire.
MOLBREEVI a reçu plusieurs désignations réglementaires, notamment Fast Track, Breakthrough Therapy et Orphan Drug Designation de la part de la FDA et de l'EMA. Le médicament a démontré des résultats positifs lors de l'essai IMPALA-2 en Phase 3, montrant un profil bénéfice-risque favorable. S'il est approuvé, MOLBREEVI serait le premier traitement approuvé pour aPAP aux États-Unis et en Europe.
Savara Inc. (Nasdaq: SVRA) hat eine schrittweise Einreichung eines Antrags auf biologische Lizenz (BLA) bei der FDA für MOLBREEVI begonnen, das auf die Behandlung der autoimmunen pulmonalen Alveolarproteinosis (aPAP), einer seltenen Lungenerkrankung, abzielt. Das Unternehmen erwartet, die BLA-Einreichung bis Ende des ersten Quartals 2025 abzuschließen und wird eine priorisierte Prüfung beantragen.
MOLBREEVI hat mehrere regulatorische Bezeichnungen erhalten, darunter Fast Track, Breakthrough Therapy und Orphan Drug Designation von sowohl der FDA als auch der EMA. Das Medikament zeigte positive Ergebnisse in der Phase-3-IMPALA-2-Studie und wies ein günstiges Verhältnis von Nutzen zu Risiko auf. Wenn es genehmigt wird, wäre MOLBREEVI die erste genehmigte Behandlung für aPAP in den USA und Europa.
- Initiated rolling BLA submission for MOLBREEVI, with completion expected by Q1 2025
- Multiple regulatory designations secured (Fast Track, Breakthrough Therapy, Orphan Drug)
- Positive Phase 3 IMPALA-2 trial results
- First-mover advantage with no currently approved treatments for aPAP in US/Europe
- Extended timeline for complete BLA submission (Q1 2025)
- No guaranteed FDA approval despite positive trial results
Insights
The initiation of a rolling BLA submission for MOLBREEVI represents a critical regulatory milestone for Savara. The Fast Track and Breakthrough Therapy designations already secured significantly expedite the review process, while the rolling submission format allows for faster completion of the application. The expected completion by Q1 2025 sets a clear timeline for potential market entry.
The multiple regulatory designations (Orphan Drug, Innovation Passport, PIM) across different jurisdictions (FDA, EMA, MHRA) strengthen MOLBREEVI's market position and potential for accelerated approval pathways. These designations typically translate to 7 years of market exclusivity in the US and 10 years in the EU upon approval.
For investors, the rolling BLA submission initiates a defined regulatory timeline that could lead to commercialization in a market with no approved treatments. The priority review request, if granted, would reduce the standard review time from 10 months to 6 months post-submission completion.
MOLBREEVI targets aPAP, a rare disease affecting the lungs' ability to process surfactant proteins - think of it as the lungs developing a "traffic jam" of proteins that should normally help with breathing. The positive Phase 3 IMPALA-2 trial results suggest the treatment could become the first FDA-approved therapy for this condition.
The market opportunity here is compelling - while rare, aPAP significantly impacts quality of life and has no approved treatments in major markets. Current management typically involves invasive whole lung lavage procedures. A targeted therapy like MOLBREEVI could revolutionize the treatment paradigm.
From an investment perspective, success in rare diseases can be highly lucrative. Orphan drugs typically command premium pricing and with no approved competitors, MOLBREEVI could capture the entire addressable market. The breakthrough therapy designation also validates the treatment's potential significant advantages over existing options.
-- Company Expects to Complete BLA Submission by End of 1Q 2025 --
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
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).
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.
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.
Forward-Looking Statements
Savara cautions you that statements in this press release that are not a description of historical fact are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as “expect,” “intend,” “plan,” “anticipate,” “believe,” and “will,” among others. Such statements include, but are not limited to, statements related to the anticipated timing of the completion of our BLA submission, the plan to request for priority review, and our belief that MOLBREEVI demonstrates a favorable benefit-risk profile and could fundamentally change the way aPAP is treated. Savara may not actually achieve any of the matters referred to in such forward-looking statements, and you should not place undue reliance on these forward-looking statements. These forward-looking statements are based upon Savara’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the risks associated with our ability to successfully develop, obtain regulatory approval for, and commercialize MOLBREEVI for aPAP; the risks and uncertainties related to the impact of widespread health concerns or changing economic or geopolitical conditions; the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations; the availability of sufficient resources for Savara’s operations and to conduct or continue planned clinical development programs; and the timing and ability of Savara to raise additional capital as needed to fund continued operations. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. For a detailed description of our risks and uncertainties, you are encouraged to review our documents filed with the SEC including our recent filings on Form 8-K, Form 10-K and Form 10-Q. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Savara undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law.
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Media and Investor Relations Contact
Savara Inc.
Temre Johnson, Executive Director, Corporate Affairs
ir@savarapharma.com
Source: Savara Inc.
FAQ
When will Savara (SVRA) complete the BLA submission for MOLBREEVI?
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