Therapy Areas: Hereditary Disorders
Molgradex Received Fast Track Designation by FDA for Treatment of Autoimmune Pulmonary Alveolar Proteinosis
8 May 2019 - - The US Food and Drug Administration granted Fast Track designation for Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), US-based orphan lung disease company Savara Inc. (NASDAQ: SVRA) said.

Molgradex, the company's lead product candidate, is being investigated in a pivotal Phase 3 study, called IMPALA, for the treatment of autoimmune pulmonary alveolar proteinosis.

Topline results from the study are expected in June 2019.

Positive results would facilitate the submission of a Biologics License Application in the first half of 2020, with an anticipated commercial launch later in 2020 or early 2021.

The Fast Track program facilitates the expedited development and review of new drugs or biologics that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.

A drug granted Fast Track designation may be eligible for Priority Review or Rolling Review of the BLA, if relevant criteria are met.

Savara is an orphan lung disease company. Savara's pipeline comprises Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis, in Phase 2a development for nontuberculous mycobacterial lung infection in both non-cystic fibrosis and CF-affected individuals with chronic NTM lung infection; and AeroVanc, a Phase 3-stage inhaled vancomycin for treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in CF.

Savara's strategy involves expanding its pipeline of potentially best-in-class products through indication expansion, strategic development partnerships and product acquisitions, with the goal of becoming a company in its field.

The most recent acquisition is aerosolized amikacin/fosfomycin, a Phase 2-ready, proprietary combination antibiotic, which has demonstrated potent and broad-spectrum antibacterial activity against highly drug resistant pathogens.

Savara's management team has significant experience in orphan drug development and pulmonary medicine, identifying unmet needs, developing and acquiring new product candidates, and effectively advancing them to approvals and commercialization.