Lifeline Delayed: FDA Hits Pause on Promising Rare Lung Disease Drug MOLBREEVI

The FDA has issued a Refusal to File (RTF) letter for MOLBREEVI, Savara's hopeful treatment for autoimmune pulmonary alveolar proteinosis (autoimmune PAP), citing needs for more manufacturing data^{1, 3, 7}.
Savara remains undeterred, expressing strong confidence and planning a swift FDA meeting to resubmit its application by Q4 2025^{1, 2}.
Crucially, the RTF does not question MOLBREEVI's safety or effectiveness, and its existing Fast Track and Breakthrough Therapy designations remain secure, sustaining hope for patients^{1, 7}.

In a challenging turn for patients battling the rare and debilitating autoimmune PAP, Savara (Nasdaq: SVRA) announced the U.S. Food and Drug Administration (FDA) has issued a Refusal to File (RTF) letter for MOLBREEVI. This decision halts, for now, the progress of a biologic therapy many hoped would soon offer relief from a condition where surfactant dangerously clogs the lungs, impairing breathing^{1, 6}.

The FDA's preliminary review of the March 2025 Biologics License Application (BLA) concluded it was "not sufficiently complete to permit substantive review," specifically requesting additional Chemistry, Manufacturing, and Controls (CMC) information¹. Importantly, the agency raised no safety concerns and did not request further efficacy studies, a silver lining in an otherwise stark announcement^{1, 7}.

Savara is moving decisively. "The requested CMC data... are currently being generated," stated Matt Pauls, Chair and CEO, expressing confidence. "We expect to resubmit our BLA in the fourth quarter of 2025"¹. The company intends to request a Type A meeting with the FDA within 30 days to align on next steps¹.

MOLBREEVI (molgramostim), an inhaled therapy, has shown significant promise, with clinical data demonstrating improved lung function and quality of life for autoimmune PAP patients^{1, 4}. The drug's journey has been marked by multiple FDA and international recognitions, including Fast Track, Breakthrough Therapy, and Orphan Drug designations, all of which remain unaffected by this RTF⁷. Savara continues its work to establish a redundant supply chain and aims for a potential launch in early 2026, contingent on eventual approval^{1, 5}.

Lifeline Delayed: FDA Hits Pause on Promising Rare Lung Disease Drug MOLBREEVI

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