The FDA has granted orphan drug status to Savara Pharmaceuticals’ AeroVanc (vancomycin hydrochloride inhalation powder) for the treatment of pulmonary methicillin-resistant Staphylococcus aureus (MRSA) infection in cystic fibrosis (CF).
AeroVanc is the first inhaled antibiotic being developed to address the growing population of MRSA-infected CF patients. Savara is currently preparing for its phase IIa clinical study of AeroVanc's efficacy, to be carried out in 20 CF centers in the U.S. In phase I studies of AeroVanc in healthy volunteers and CF patients, AeroVanc was well tolerated and demonstrated an excellent pharmacokinetic profile.
"AeroVanc is a much needed addition to the current treatment options for MRSA infected CF patients," said Michael Konstan, M.D., chairman, department of pediatrics, and director of the Cystic Fibrosis Center at Rainbow Babies and Children's Hospital and Case Western Reserve University. "In the absence of an FDA approved inhaled antibiotic therapy directed at MRSA, CF clinicians are increasingly prescribing off-label nebulization of the intravenous formulation of vancomycin. This therapy is generally well tolerated, has high antibacterial activity against MRSA, and patients have a good clinical response."
Robert Neville, CEO of Savara Pharmaceuticals, said, "Orphan drug designation in combination with our intellectual property provides strong market exclusivity potential for AeroVanc. We hope to provide similar benefits to MRSA infected patients that tobramycin provides for the treatment of Pseudomonas aeruginosa infections. The response from the cystic fibrosis community has been very encouraging, so much so that physicians like to refer to AeroVanc as the 'TOBI for MRSA'."
Orphan drug designation qualifies a company for several benefits, including the potential for market exclusivity, development grants and for certain tax credits.