Austin, TX, November 13, 2012 – Savara Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to AeroVancTM (vancomycin hydrochloride inhalation powder) for the treatment of pulmonary methicillin-resistant Staphylococcus aureus (MRSA) infection in cystic fibrosis (CF) patients. Orphan drug designation qualifies a company for several benefits, including the potential for market exclusivity, development grants, and for certain tax credits.
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 United States. 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.”
“Orphan drug designation in combination with our intellectual property provides strong market exclusivity potential for AeroVanc,” said Robert Neville, Chief Executive Officer of Savara Pharmaceuticals. “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’ .”
The U.S. Orphan Drugs Act aims to encourage the development of drugs involved in the diagnosis, prevention or treatment of a medical condition affecting fewer than 200,000 people in the United States. Orphan drug designation grants U.S. market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA specifications. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA user fee, and the orphan status also allows the sponsor to apply for grants to support clinical trials.
AeroVanc™ (vancomycin hydrochloride inhalation powder) is a proprietary inhaled dry powder form of vancomycin in a capsule-based device designed for convenient self-administration. AeroVanc is being developed for the treatment of MRSA lung infection in cystic fibrosis patients. Vancomycin administered by IV is the antibiotic of choice for the treatment of MRSA-related bronchopneumonia, however, IV administration, poor penetration into the lungs and systemic toxicities limit its use in a chronic setting. By delivering vancomycin directly to the site of infection, AeroVanc is expected to improve clinical efficacy and reduce adverse effects due to systemic drug exposure.
AeroVanc has demonstrated positive safety and tolerability results in Phase I clinical studies conducted in healthy subjects and patients with cystic fibrosis, with a pharmacokinetic profile that supports its potential as a once- or twice-daily treatment for pulmonary MRSA infection.
About Cystic Fibrosis and MRSA
Cystic fibrosis is a life-shortening genetic disease characterized by thick, sticky mucus in the lungs and frequent lung infections, which result in loss of lung function. As the disease progresses, the lungs of CF patients are typically infected with bacteria that are difficult to eradicate. Such infections are usually treated with inhaled antibiotics. In recent years, infection by MRSA has become increasingly common, with a prevalence of almost 30 percent of the estimated 30,000 CF patients in the United States. Persistent MRSA infection is associated with faster decline in lung function and a significantly shortened life expectancy. Currently there is no approved inhaled therapy for MRSA infection for CF patients.
About Savara Pharmaceuticals
Savara Pharmaceuticals is an emerging specialty pharmaceutical company developing innovative pulmonary drugs for the treatment of serious and life-threatening conditions. The company’s lead product, AeroVanc™ (vancomycin hydrochloride inhalation powder), is the first dry powder inhaled antibiotic for the treatment of methicillin-resistant Staphylococcus aureus (MRSA) infection in patients with cystic fibrosis. For more information, please see Savara’s website at www.savarapharma.com.
Robert Neville, CEO