As it prepares to submit its molgramostim inhalation solution for FDA approval to treat autoimmune pulmonary alveolar proteinosis (aPAP), Savara is laying some groundwork in raising awareness of the rare disease.
The Texas-based biotech sponsored a recent episode of “The Balancing Act,” a morning talk show that airs on Lifetime. Per an announcement from the company Tuesday, the episode included a segment—part of the show’s recurring “Behind the Mystery” series highlighting the stories of patients with rare and genetic diseases—focusing specifically on aPAP.
Pulmonary alveolar proteinosis is caused by a buildup in the lungs’ alveoli of a substance called surfactant that’s made up of proteins and lipids. The buildup can block air from moving through the lungs and into the blood, making it difficult to breathe and, in severe cases, leading to respiratory failure. Autoimmune PAP is the most common form of the disease, in which the immune system erroneously attacks the cells tasked with clearing the alveoli.
The nine-minute segment features interviews with and follows an aPAP patient, Eric, and Bruce Trapnell, M.D., who’s a pulmonologist and also the international coordinating investigator for Savara’s ongoing IMPALA-2 phase 3 clinical trial of molgramostim in aPAP. Throughout the piece, Eric and Trapnell discuss barriers to diagnosis and treatment of aPAP as well as what it’s like to live with the disease.
“We are pleased to partner with The Balancing Act to highlight aPAP, a rare respiratory disease with no currently approved medicines in the United States or Europe and a high unmet need for patients,” Savara CEO Matt Pauls said in the announcement. “Educating people about the hallmark signs and symptoms of aPAP, as well as the simple, accurate, non-invasive blood test to help diagnose the disease, may increase awareness for this debilitating condition and provide a quicker path to diagnosis for patients.”
Savara is planning to wrap up its FDA biologics license application for molgramostim as an aPAP treatment in the first half of next year; the therapeutic has already earned orphan drug, fast track and breakthrough therapy designations from the agency.
Molgramostim is a granulocyte-macrophage colony-stimulating factor designed to aid in clearing surfactant from the alveoli in aPAP patients. It’s delivered via Pari Pharma’s eFlow nebulizer technology.
The company announced earlier this year that the IMPALA-2 trial had met its primary endpoint and multiple secondary endpoints. For the former, once-daily inhalation of a 300-mcg dose of molgramostim was found to produce a statistically significant improvement in the diffusing capacity of the lungs for carbon monoxide—a measure known as DLCO, representing how well the lungs can transfer carbon dioxide from inhaled air into the blood—compared to placebo after 24 weeks. The improvements were then shown to last through at least Week 48, supporting one of the study’s secondary endpoints.