Savara Announces Expanded Access Program (EAP) for Molgramostim Inhalation Solution (Molgramostim) for Patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Savara Inc. (Nasdaq: SVRA) has announced the Savara Early Access Program, an Expanded Access Program (EAP) for molgramostim inhalation solution to treat patients with autoimmune pulmonary alveolar proteinosis (aPAP). This program allows physicians to request molgramostim for eligible aPAP patients in select regions where the product is not commercially available, in compliance with local regulations.
The EAP has been approved by the U.S. Food and Drug Administration (FDA) and is currently accepting requests from eligible patients in select North American and European countries, with plans to expand through 2026. Savara plans to submit a Biologics License Application to the FDA for molgramostim in aPAP in the first half of 2025.
Molgramostim has received Orphan Drug, Fast Track, and Breakthrough Therapy designations from the FDA, as well as similar designations from European and UK regulatory agencies for the treatment of aPAP.
Savara Inc. (Nasdaq: SVRA) ha annunciato il Savara Early Access Program, un Expanded Access Program (EAP) per la soluzione di inalazione di molgramostim per trattare pazienti con proteinosi alveolare autoimmunitaria (aPAP). Questo programma consente ai medici di richiedere molgramostim per pazienti aPAP idonei in selezionate regioni dove il prodotto non è disponibile commercialmente, in conformità con le normative locali.
L'EAP è stato approvato dalla Food and Drug Administration (FDA) degli Stati Uniti e sta attualmente accettando richieste da pazienti idonei in alcuni paesi del Nord America e dell'Europa, con piani di espansione fino al 2026. Savara prevede di presentare una Domanda di Licenza Biologica alla FDA per molgramostim in aPAP nella prima metà del 2025.
Molgramostim ha ricevuto le designazioni di Farmaco Orfano, Fast Track e Terapia Innovativa dalla FDA, così come designazioni simili da agenzie regolatorie europee e del Regno Unito per il trattamento della aPAP.
Savara Inc. (Nasdaq: SVRA) ha anunciado el Savara Early Access Program, un Expanded Access Program (EAP) para la solución de inhalación de molgramostim para tratar a pacientes con proteinosis alveolar autoinmunitaria (aPAP). Este programa permite que los médicos soliciten molgramostim para pacientes elegibles de aPAP en regiones seleccionadas donde el producto no está disponible comercialmente, cumpliendo con las regulaciones locales.
El EAP ha sido aprobado por la Administración de Alimentos y Medicamentos (FDA) de EE. UU. y actualmente está aceptando solicitudes de pacientes elegibles en algunos países de América del Norte y Europa, con planes de expansión hasta 2026. Savara planea presentar una Solicitud de Licencia Biológica ante la FDA para molgramostim en aPAP en la primera mitad de 2025.
Molgramostim ha recibido las designaciones de Medicamento Huérfano, Fast Track y Terapia Innovadora de la FDA, así como designaciones similares de agencias reguladoras europeas y del Reino Unido para el tratamiento de aPAP.
Savara Inc. (Nasdaq: SVRA)는 자가면역 폐포 단백증(aPAP) 치료를 위한 몰그라마스팀 흡입 용액의 조기 접근 프로그램(Savara Early Access Program)인 확대 접근 프로그램(EAP)을 발표했습니다. 이 프로그램은 의사들이 상업적으로 이용할 수 없는 특정 지역에서 자격이 있는 aPAP 환자에게 몰그라마스팀을 요청할 수 있도록 허용합니다.
EAP는 미국 식품의약국(FDA)의 승인을 받았으며, 현재 북미 및 유럽의 일부 국가에서 자격이 있는 환자의 요청을 받고 있으며, 2026년까지 확대할 계획입니다. Savara는 2025년 상반기에 aPAP에 대한 몰그라마스팀의 생물학적 라이센스 신청을 FDA에 제출할 계획입니다.
몰그라마스팀은 aPAP 치료를 위한 희귀약, 패스트트랙 및 혁신 치료 지정을 FDA로부터 받았으며, 유럽 및 영국의 규제 기관에서도 유사한 지정을 받았습니다.
Savara Inc. (Nasdaq: SVRA) a annoncé le programme d'accès anticipé Savara, un Expanded Access Program (EAP) pour la solution inhalée de molgramostim destinée à traiter les patients atteints de protéinoses alvéolaires auto-immunes (aPAP). Ce programme permet aux médecins de demander du molgramostim pour des patients aPAP éligibles dans certaines régions où le produit n'est pas disponible commercialement, conformément aux réglementations locales.
L'EAP a été approuvé par la Food and Drug Administration (FDA) des États-Unis et accepte actuellement les demandes de patients éligibles dans certains pays d'Amérique du Nord et d'Europe, avec des plans d'expansion jusqu'en 2026. Savara prévoit de soumettre une demande de licence biologique à la FDA pour le molgramostim dans l'aPAP au premier semestre 2025.
Le molgramostim a reçu des désignations de médicament orphelin, de Fast Track et de thérapie révolutionnaire de la FDA, ainsi que des désignations similaires d'agences réglementaires européennes et britanniques pour le traitement de l'aPAP.
Savara Inc. (Nasdaq: SVRA) hat das Savara Early Access Program angekündigt, ein Expanded Access Program (EAP) für die Inhalationslösung von Molgramostim zur Behandlung von Patienten mit autoimmuner alveolärer Proteinose (aPAP). Dieses Programm ermöglicht es Ärzten, Molgramostim für berechtigte aPAP-Patienten in ausgewählten Regionen anzufordern, in denen das Produkt nicht kommerziell erhältlich ist, und erfüllt die lokalen Vorschriften.
Das EAP wurde von der U.S. Food and Drug Administration (FDA) genehmigt und akzeptiert derzeit Anfragen von berechtigten Patienten in bestimmten nordamerikanischen und europäischen Ländern, mit Plänen zur Expansion bis 2026. Savara plant, in der ersten Hälfte des Jahres 2025 einen Biologics License Application bei der FDA für Molgramostim in aPAP einzureichen.
Molgramostim hat von der FDA die Orphan Drug, Fast Track und Breakthrough Therapy Bezeichnungen erhalten sowie ähnliche Bezeichnungen von europäischen und britischen Regulierungsbehörden zur Behandlung von aPAP.
- Expanded Access Program (EAP) approved by FDA for molgramostim in aPAP treatment
- EAP available in select North American and European countries, with plans to expand through 2026
- Positive results demonstrated in Phase 3 IMPALA-2 clinical trial
- Biologics License Application submission planned for first half of 2025
- Molgramostim granted multiple regulatory designations (Orphan Drug, Fast Track, Breakthrough Therapy) by FDA, EMA, and UK MHRA
- None.
Insights
The announcement of Savara's Expanded Access Program (EAP) for molgramostim is a significant development for patients with autoimmune pulmonary alveolar proteinosis (aPAP). This program allows pre-approval access to the investigational drug in select regions where it's not commercially available, addressing a critical unmet need in aPAP treatment.
The EAP's approval by the FDA underscores the potential efficacy of molgramostim, especially considering the positive results from the Phase 3 IMPALA-2 trial. The multiple designations granted to molgramostim, including Orphan Drug, Fast Track and Breakthrough Therapy, highlight its promise in treating this rare lung disease.
For investors, this development signals potential market expansion and early revenue generation before full commercial approval. The planned BLA submission in H1 2025 provides a clear timeline for potential FDA approval, which could be a significant catalyst for Savara's stock.
The initiation of the EAP for molgramostim represents a strategic move by Savara to establish market presence and gather real-world data before full commercial launch. This approach can help build brand recognition and physician familiarity with the product, potentially accelerating market uptake post-approval.
The program's expansion plans through 2026 suggest a long-term commitment to patient access and market development. For investors, this indicates a well-thought-out commercialization strategy that could translate into sustained revenue growth in the rare disease space.
Given Savara's
Program Enables Physicians to Request Molgramostim for Eligible Patients in Select Geographies Where the Product is Not Commercially Available and in Compliance with Local Regulatory Requirements
The Savara Early Access Program has been reviewed and allowed to proceed by the
"Expanded access is granted to investigational products that address a serious condition for which there are no comparable therapies available," said Matt Pauls, Chair and CEO of Savara. "Given the high unmet need in aPAP, and positive results demonstrated in the Phase 3 IMPALA-2 clinical trial, we felt it was critically important to establish the Savara Early Access Program to allow eligible aPAP patients pre-approval access to molgramostim. This program reflects our ongoing commitment to the global aPAP community and the goal of potentially delivering an effective therapy for patients with this rare lung disease as quickly as possible.”
Savara plans to complete submission of a Biologics License Application to the FDA for molgramostim in aPAP in the first half of 2025. Molgramostim has been granted Orphan Drug, Fast Track, and Breakthrough Therapy designations from the FDA, Orphan Drug designation from the European Medicines Agency and Innovative Passport and Promising Innovative Medicine designation from the
About Expanded Access to Molgramostim
EAPs are intended to serve as a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical treatment outside of clinical trials before it is commercially available, when no comparable or satisfactory alternative therapy options are available. Healthcare professionals and autoimmune Pulmonary Alveolar Proteinosis (aPAP) patients who are interested to learn more about Savara's EAP for molgramostim, including eligibility criteria, may visit www.clinicaltrials.gov, NCT06546098, or contact medicalinfo@savarapharma.com.
About aPAP
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 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.
About Savara
Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim inhalation solution, is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (aPAP). Molgramostim 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, formerly known as Twitter: @SavaraPharma, LinkedIn: www.linkedin.com/company/savara-pharmaceuticals/).
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 future expansion of the Savara Early Access Program, Savara’s goal of potentially delivering an effective therapy as rapidly as possible, and the anticipated timing of Savara’s BLA submission. 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 molgramostim for aPAP; the actions and decisions of regulatory authorities; 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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Savara Inc. IR & PR
Anne Erickson (anne.erickson@savarapharma.com)
(512) 851-1366
Source: Savara Inc.
FAQ
What is the Savara Early Access Program for molgramostim?
When does Savara (SVRA) plan to submit a Biologics License Application for molgramostim?
What regulatory designations has molgramostim received for aPAP treatment?
In which countries is the Savara Early Access Program for SVRA's molgramostim currently available?
