Chronic respiratory diseases were the third-leading cause of global deaths in 2019 and can be caused by infection, smoking tobacco, radon, asbestos or other forms of air pollution.
Affecting around one in every 150,000 people in the US, aPAP is characterised by myeloid cell dysfunction, abnormal pulmonary surfactant accumulation and innate immune deficiency.
The trial met its primary endpoint after the therapy improved lung function in haemoglobin-adjusted percent predicted DLCO, a test that measures the diffusing capacity of the lung for carbon monoxide, achieving statistical significance compared to placebo at 24 weeks, which was maintained at 48 weeks.
Bruce Trapnell, lead investigator of the trial, commented: “With convincing data from two large clinical trials, the evidence now clearly demonstrates molgramostim has the potential to be a safe and efficacious treatment option for [aPAP] patients.”
Matt Pauls, chair and chief executive officer, Savara, said: “The strong efficacy data and favourable benefit-risk profile potentially positions molgramostim to be the first and only approved therapeutic for aPAP in the US and Europe.”
The inhaled form of recombinant human granulocyte-macrophage colony-stimulating factor has already been granted Orphan Drug, Fast Track and Breakthrough Therapy designations by the US Food and Drug Administration (FDA), Orphan Drug Designation from the European Medicines Agency (EMA), as well as the Innovative Passport and Promising Innovative Medicine designations from the UK’s Medicines and Healthcare Products Regulatory Agency for the treatment of aPAP.