The newly FDA approved inhalational tobramycin TOBI Podhaler produced by Novartis is being hailed by the cystic fibrosis community and drug makers like Texas-based Savara Pharmaceuticals.
The TOBI Podhaler has been recently approved by US Food and Drug Administration and is a potent antibiotic therapy for the management of lung infections caused by Pseudomonas aeruginosa (the most common pathogen in cystic fibrosis patients) that is refractory to most antibiotic regimens. The CEO of Savara Pharmaceuticals welcomed the approval, stating that he is “delighted that the FDA has approved a very necessary product.”
Researchers at Savara Pharmaceuticals have long been working to devise a therapy to treat another commonly reported pathogen that can cause severe lung infection in CF patients, i.e. methicillin-resistant Staphylococcus aureus that has the capability to inhibit the lower respiratory tract.
Cystic Fibrosis is marked by production of thick mucus plugs due to inherited deficiency of genes coding for the chloride transporter channels. Due to defects in the channels, respiratory secretions tend to be thicker and can clog respiratory passage-ways and act as breeding grounds for aerobic and anaerobic pathogens, leading to mixed infections that are hard to treat and may become a cause of overt respiratory failure or even death in severe cases.
MRSA (methicillin-resistant Staphylococcus aureus) is associated with a poor prognosis and an ever-higher rate of complications in CF patients.
Savara Pharmaceuticals’ AeroVanc is the company’s leading antibiotic product used to treat CF patients, and contains vancomycin in the form of dry powder formulation that can be introduced via intravenous route. AeroVanc is preferred over Vancomycin on the basis of it offering more optimal therapeutic and side effect profiles.
Researchers at Savara Pharmaceuticals are aiming to decrease the systemic side effects of vancomycin by introduction of active drug molecules directly in the lungs. The capsular device of AeroVanc can be administered once or twice daily for the management of MRSA infection.
Savara Pharmaceuticals is very hopeful after the promising results of Phase 1 clinical trial of AeroVanc and similar degree of efficacy is expected from the upcoming phase 2a clinical trials. According to CEO Rob Neville of Savara Pharmaceuticals, 80 CF patients (with chronic MRSA infection) will be enrolled in the phase 2a study and the participants will be chosen from the U.S. and Canada. To finance the study, Savara Pharmaceutical has managed to cross the second tranche of $16 million in funding.
The additional funding of $4.6 million has been received from the Texas Emerging Technology Fund, the Austin Technology Incubator, the National Institutes of Health and Qualifying Therapeutic Discovery Program.
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