Savara Inc., an orphan lung disease company, announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of aPAP.
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).
The Molgradex Breakthrough Therapy designation is based on data from IMPALA, a pivotal Phase 3 clinical study evaluating Molgradex for the treatment of aPAP. Data from the study was recently presented in an oral session at the 2019 European Respiratory Society (ERS) International Congress in Madrid, Spain.
“We are pleased that the FDA recognized Molgradex as a breakthrough therapy for the treatment of aPAP, a debilitating rare lung disease with no approved pharmacologic treatment options,”
said Rob Neville, Chief Executive Officer, Savara.
“We believe this designation reflects the significance of Molgradex as an investigational product that, based on the IMPALA study, has been demonstrated to improve patient outcomes. The breakthrough designation is designed to provide increased collaboration and more frequent dialogue with the FDA and is an important milestone as we work to determine the best path forward for this product.”