New Hope for PAP: Savara Taps PANTHERx for MOLBREEVI Launch

LANGHORNE, PA – January 08, 2026 – In a strategic move poised to reshape the treatment landscape for a debilitating rare lung disease, clinical-stage biopharmaceutical company Savara Inc. has named PANTHERx® Rare Pharmacy as the exclusive U.S. specialty pharmacy partner for its investigational therapy, MOLBREEVI. The announcement comes as Savara awaits a decision from the U.S. Food and Drug Administration (FDA) on its Biologics License Application (BLA) for the drug, which seeks approval to treat autoimmune Pulmonary Alveolar Proteinosis (autoimmune PAP).

This exclusive agreement signals a proactive, patient-centric approach to commercialization, establishing a comprehensive support and distribution framework well ahead of a potential FDA approval. For the small community of patients battling autoimmune PAP, this partnership represents more than just a new drug in the pipeline; it signifies a commitment to a holistic care model designed to ease the immense burdens of living with a chronic, rare condition.

A Breakthrough for a Debilitating Lung Disease

Autoimmune PAP is a rare, life-altering disease that affects an estimated 7 to 10 people per million. It occurs when the body’s immune system mistakenly creates antibodies that neutralize a critical protein called granulocyte-macrophage colony-stimulating factor (GM-CSF). In a healthy lung, GM-CSF signals immune cells known as alveolar macrophages to clear excess surfactant—a fatty substance that lines the air sacs (alveoli) and prevents them from collapsing. When this signaling is blocked, surfactant builds up, clogging the alveoli and severely impairing the lungs' ability to transfer oxygen to the blood.

Patients often endure a grueling diagnostic odyssey, with non-specific symptoms like progressive shortness of breath, chronic cough, and profound fatigue frequently misdiagnosed as more common conditions like pneumonia. On average, it can take 18 months from a patient's first physician visit to receive an accurate diagnosis. The long-term consequences can be severe, including pulmonary fibrosis, respiratory failure, and the need for a lung transplant.

The current standard of care is whole lung lavage (WLL), a highly invasive procedure performed under general anesthesia where a patient's lungs are repeatedly flushed with saline to mechanically wash out the accumulated surfactant. While effective for some, WLL requires hospitalization and can be a physically and emotionally taxing ordeal that patients may need to undergo repeatedly.

MOLBREEVI offers a new paradigm. As an inhaled, recombinant form of human GM-CSF, it is designed to directly address the root cause of the disease. Delivered via a proprietary eFlow® Nebulizer System, the therapy aims to replenish the functional GM-CSF in the lungs, restoring the ability of macrophages to clear surfactant. The goal is to improve oxygenation and lung function, potentially offering patients the first-ever approved, targeted pharmacological treatment for their condition.

Navigating the Rigorous Path to Approval

MOLBREEVI's journey to market has been closely watched. Savara resubmitted its BLA to the FDA in December 2025, having diligently addressed prior agency feedback related to Chemistry, Manufacturing, and Controls (CMC) data. With the resubmission, the company also requested Priority Review, a designation that, if granted, would shorten the FDA's review timeline to a six-month target.

The potential for an accelerated review is bolstered by a series of significant regulatory milestones MOLBREEVI has already achieved. The FDA has granted it Breakthrough Therapy and Fast Track designations, which are reserved for drugs that may demonstrate substantial improvement over available therapy for serious conditions. Furthermore, it holds Orphan Drug designation in both the United States and Europe, acknowledging its importance for a rare disease population with high unmet medical needs. These designations collectively underscore the regulatory recognition of autoimmune PAP's severity and the urgent need for novel, effective treatments.

More Than a Medicine: A New Model for Patient Care

The partnership between Savara and PANTHERx is built on the philosophy that effective treatment for a rare disease requires more than just dispensing a medication. It demands an integrated support system that addresses the multifaceted challenges patients face.

“Our selection of PANTHERx as the exclusive U.S. specialty pharmacy for MOLBREEVI underscores Savara’s commitment to delivering more than a medicine—we will be delivering a complete care experience for patients,” said Braden Parker, Chief Commercial Officer at Savara. “This partnership reflects our shared mission to improve the lives of patients with autoimmune PAP by making their journey to therapy as smooth and supportive as possible.”

PANTHERx Rare is uniquely positioned to fulfill this mission. As the nation's largest independent rare pharmacy, it has built its reputation on a hyper-personalized approach. Its proprietary RxARECARE® model provides each patient with a dedicated, medication-specific care team that handles everything from navigating complex insurance and financial assistance programs to providing clinical case management and ongoing therapy education. This high-touch model has proven remarkably effective. PANTHERx is an eight-time winner of the prestigious MMIT Patient Choice Award for patient satisfaction and holds dual accreditations in rare disease from both the Accreditation Commission for Health Care (ACHC) and the Utilization Review Accreditation Commission (URAC).

“We are proud to partner with Savara to support the launch of MOLBREEVI and help ensure eligible patients with autoimmune PAP receive timely access and ongoing engagement throughout their treatment journey,” said Bansi Nagji, CEO of PANTHERx. He noted the pharmacy’s proven success, stating, “95.45% of the patients served by PANTHERx have no unresolved barriers at their annual assessment, and we are committed to delivering that same level of success to this community.”

The Strategic Blueprint for a Successful Rare Disease Launch

From a business perspective, Savara's decision to forge an exclusive partnership with a specialized pharmacy is a shrewd strategic move that helps de-risk the complex process of a rare disease drug launch. The autoimmune PAP market is small and highly specialized, requiring a nuanced approach that mass-market distribution channels cannot provide. By embedding PANTHERx into its commercialization plan pre-approval, Savara ensures that the intricate logistics of patient identification, onboarding, and ongoing support are managed by an expert team from day one.

This collaboration is a cornerstone of Savara’s broader commercial strategy, which includes expanding awareness of autoimmune PAP among pulmonologists, improving diagnostic pathways, and building the internal capabilities needed for a successful launch. The partnership effectively creates a turnkey solution for patient access and support, allowing Savara to focus on clinical engagement and market education while PANTHERx manages the critical patient journey. This integrated infrastructure is designed to optimize therapy adherence and persistence, which are vital for achieving the best possible clinical outcomes for individuals living with this chronic disease.