The PAP Foundation, a US organization that advocates for pulmonary alveolar proteinosis (PAP) patients, says that it supports FDA approval of Savara’s Molgradex inhaled recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF). The group said that it expressed this opinion at the FDA’s Rare Disease Day in February 2020 and is officially submitting its statement and comments from the public meeting.
In October 2019, Savara said that the FDA had discouraged the company from submitting a BLA for Molgradex for the treatment of PAP at that time. Two months later, the agency granted orphan drug designation to Molgradex for automimmune PAP in December 2019.
In June 2019, Savara announced that the Phase 3 IMPALA study of Molgradex for PAP failed to meet its primary endpoint. The foundation, however, says that it has heard numerous positive reports from patients who participated in that study and a Japanese study of inhaled GM-CSF. In addition, the group says, some patients who had experienced improvement in their condition using inhaled GM-CSF found that their condition deteriorated after discontinuing the drug.
The PAP Foundation states that, “Based on the above trials and on the experiences of numerous PAPF patients, it is our continued reasoned position that inhaled GM-CSF treatment is not only an effective therapy for many patients, but the best medical option currently available to improve the overall health and quality of life for most aPAP patients. In contrast, the current standard of care for aPAP treatment is Whole Lung Lavage (“WLL”); a highly invasive, time-consuming and dangerous surgical procedure that is often needed throughout a PAP patient’s life whenever their symptoms become life-threatening or otherwise too much to bear.”
The statement suggests that the inhaled GM-CSF therapy requires higher doses and a longer treatment period in order to produce significant improvement in PAP patients. In a recent business update, Savara said that results from the IMPALA open label follow up study demonstrated continued improvement in patients who had been treated with Molgradex long term, and it is working with the FDA to design a new Phase 3 study.
Savara CEO Rob Neville said, “Results from the open-label period of IMPALA reaffirm our strong belief that Molgradex could improve outcomes for patients with aPAP, a rare and debilitating lung disease. We continue our conversations with the FDA, as our highest priority is to get clarity from the agency regarding the design and endpoints of a second Phase 3 study in aPAP.”
Read the PAP Foundation statement.
Read the Savara press release.
