Savara Inc. (Nasdaq: SVRA) has announced the launch of its Savara Early Access Program, an Expanded Access Program (EAP) for molgramostim inhalation solution, aimed at treating patients with autoimmune pulmonary alveolar proteinosis (aPAP). This initiative allows physicians to request molgramostim for eligible aPAP patients in specific regions where the drug is not yet commercially available, adhering to local regulatory requirements.
The FDA has reviewed and approved the Savara Early Access Program, which is now open to requests from eligible patients in select countries across North America and Europe. Plans are in place to broaden the program's reach through 2026.
Addressing Unmet Needs in aPAP Treatment
According to Matt Pauls, Chair and CEO of Savara, expanded access is granted to investigational products that address serious conditions lacking comparable therapies. He stated, "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."
Regulatory Pathway and Designations
Savara is preparing to submit a Biologics License Application (BLA) to the FDA for molgramostim in aPAP during the first half of 2025. Molgramostim has already been granted Orphan Drug, Fast Track, and Breakthrough Therapy designations by the FDA. Additionally, it has received Orphan Drug designation from the European Medicines Agency and Innovative Passport and Promising Innovative Medicine designation from the UK's Medicines and Healthcare Products Regulatory Agency for aPAP treatment.
Understanding Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Autoimmune PAP is a rare lung disorder characterized by an abnormal accumulation of surfactant in the alveoli of the lungs. This buildup impairs gas exchange, leading to symptoms such as shortness of breath, cough, and fatigue. In a healthy lung, alveolar macrophages clear excess surfactant, a process stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF). However, in aPAP, antibodies neutralize GM-CSF, preventing macrophages from effectively clearing surfactant.
Accessing the Expanded Access Program
Healthcare professionals and aPAP patients seeking more information about Savara's EAP for molgramostim, including eligibility criteria, can visit www.clinicaltrials.gov (NCT06546098) or contact medicalinfo@savarapharma.com.
