Cystic Fibrosis - Liposomal Tobramycin Receives Second Orphan Drug Designation Within Weeks
Axentis Pharma (Switzerland) announced today that the Office of OrphanProducts Development of the US Food and Drug Administration (FDA) has granteda second orphan drug designation to its lead product candidateFluidosomes(TM)-tobramycin. This drug is a liposomal formulation oftobramycin and an innovative treatment for infections of the respiratorytract in patients with cystic fibrosis. Only three months ago, the FDAgranted Fluidosomes(TM)-tobramycin orphan drug designation for the treatmentof pulmonary infections caused by Pseudomonas aeruginosa. The newly grantedsecond designation relates to pulmonary infections caused by Burkholderiacepacia (B. cepacia) pathogens.
Despite stringent infection control practices, B. cepacia infectionsstill occur in cystic fibrosis patients and can lead to fatal sepsis. Thecell envelopes of these especially virulent bacteria are impermeable to mostantibiotics, which makes them particularly difficult to treat. Due to itsunique mode of action, which allows the antibiotics to penetrate into thebacteria, Fluidosomes(TM)-tobramycin could become a particularly effectivetreatment for B. cepacia infections.
Prof. Dr. Miguel A Valvano, MD, Medical Advisor to Axentis Pharma,comments on the development: "Burkholderia cepacia is almost alwaysmulti-resistant to antibiotics and this, in conjunction with the poorprognosis of patients with B. cepacia infection, makes the treatment of thesepatients exceedingly complex. Tobramycin is in principle an effectiveantibiotic. The drug is however rather ineffective due to the impermeabilityof B. cepacia's cell envelope. In addition, B. cepacia - just like many otherpathogens - has developed mechanisms to eliminate antibiotics once they haveentered the cell. Fluidosomes(TM)-tobramycin seems to overcome theselimitations by packing tobramycin into liposomes, which, by allowingeffective penetration of the antibiotic into the bacterial cell, completelychanges the microbiological profile of this antibiotic. Hence,Fluidosomes(TM)-tobramycin could be a totally new antibiotic formulation thataddresses microbiological needs that no other antibiotic can."
What exactly happens when Fluidosomes(TM)-tobramycin encounters thebacterium is still not entirely clear, but pre-clinical data indicate a novelmode of action. Dr. Helmut Brunar, CEO of Axentis Pharma explains: "Once atthe site of infection, tobramycin-containing liposomes seem to fuse with thecell membrane of the pathogen. In this way, the entire load of tobramycincontained in the Fluidosomes(TM) is released into the bacterial cell.Additionally, our data indicate that bacterial rescue mechanisms that pumptobramycin out of the cell are inhibited by the fusion process. The efficientdelivery and maximum release of tobramycin into the bacterial cell togetherwith inhibition of the clearance mechanism indicate thatFluidosomes(TM)-tobramycin has a highly efficient therapeutic effect."
About Axentis Pharma AG (http://www.axentispharma.com)
Axentis Pharma is a respiratory specialty pharmaceutical company whosecore competence is the combination of a fully patented, liposome-based drugdelivery system with already established and well-characterized therapeuticagents. The company is using its platform delivery technology, namedFluidosomes(TM) technology, for the development of its lead product, aninhalable liposomal formulation of tobramycin. Axentis Pharma's lead productis designed to treat bacterial infections in the lungs.
About Fluidosomes(TM) technology
Axentis Pharma's Fluidosomes(TM) technology uses biocompatible lipidsendogenous to the lung that are formulated into small liposomes. Thisnanocapsule platform offers wide-ranging potential for unmet medical needs,including chronic respiratory infections of the lung. In the case ofFluidosomes(TM)-tobramycin, the interaction between tobramycin and themicrobial cell is triggered when the liposomes undergo a fusion process withthe outer membrane of the bacterial cell wall. Tobramycin then penetratesinto the inner cell compartment and triggers bacterial cell death.For further information, please contact: Dr. Helmut Brunar, Ph.D., CEO Axentis Pharma AG Limmatquai 138 8001 Zurich, Switzerland T +41-44-202-7878 E firstname.lastname@example.org W http://www.axentispharma.com Copy Editing & Distribution: PR&D - Public Relations for Research & Education Campus Vienna Biocenter 2 1030 Vienna, Austria T +43-1-505-70-44 E email@example.com W http://www.prd.at
SOURCE Axentis Pharma AG
You May Also Like