Navigation Links
Cystic Fibrosis - Liposomal Tobramycin Receives Second Orphan Drug Designation Within Weeks
Date:7/16/2009

ZURICH, July 16 /PRNewswire/ -- An innovative treatment for infections of the respiratory tract in cystic fibrosis patients has received a second orphan drug designation in the US only weeks after a first designation was granted. The recent designation relates to Burkholderia cepacia pathogens that can cause lethal infections in cystic fibrosis patients. For Axentis Pharma AG of Zurich, Switzerland, both designations affirm the therapeutic potential of its product candidate Fluidosomes(TM)-tobramycin, whose unique microbiological profile sets it apart from other antibiotic formulations (including free tobramycin).

Axentis Pharma (Switzerland) announced today that the Office of Orphan Products Development of the US Food and Drug Administration (FDA) has granted a second orphan drug designation to its lead product candidate Fluidosomes(TM)-tobramycin. This drug is a liposomal formulation of tobramycin and an innovative treatment for infections of the respiratory tract in patients with cystic fibrosis. Only three months ago, the FDA granted Fluidosomes(TM)-tobramycin orphan drug designation for the treatment of pulmonary infections caused by Pseudomonas aeruginosa. The newly granted second designation relates to pulmonary infections caused by Burkholderia cepacia (B. cepacia) pathogens.

Despite stringent infection control practices, B. cepacia infections still occur in cystic fibrosis patients and can lead to fatal sepsis. The cell envelopes of these especially virulent bacteria are impermeable to most antibiotics, which makes them particularly difficult to treat. Due to its unique mode of action, which allows the antibiotics to penetrate into the bacteria, Fluidosomes(TM)-tobramycin could become a particularly effective treatment for B. cepacia infections.

Prof. Dr. Miguel A Valvano, MD, Medical Advisor to Axentis Pharma, comments on the development: "Burkholderia cepacia is almost always multi-resistant to antibiotics and this, in conjunction with the poor prognosis of patients with B. cepacia infection, makes the treatment of these patients exceedingly complex. Tobramycin is in principle an effective antibiotic. The drug is however rather ineffective due to the impermeability of B. cepacia's cell envelope. In addition, B. cepacia - just like many other pathogens - has developed mechanisms to eliminate antibiotics once they have entered the cell. Fluidosomes(TM)-tobramycin seems to overcome these limitations by packing tobramycin into liposomes, which, by allowing effective penetration of the antibiotic into the bacterial cell, completely changes the microbiological profile of this antibiotic. Hence, Fluidosomes(TM)-tobramycin could be a totally new antibiotic formulation that addresses microbiological needs that no other antibiotic can."

What exactly happens when Fluidosomes(TM)-tobramycin encounters the bacterium is still not entirely clear, but pre-clinical data indicate a novel mode of action. Dr. Helmut Brunar, CEO of Axentis Pharma explains: "Once at the site of infection, tobramycin-containing liposomes seem to fuse with the cell membrane of the pathogen. In this way, the entire load of tobramycin contained in the Fluidosomes(TM) is released into the bacterial cell. Additionally, our data indicate that bacterial rescue mechanisms that pump tobramycin out of the cell are inhibited by the fusion process. The efficient delivery and maximum release of tobramycin into the bacterial cell together with inhibition of the clearance mechanism indicate that Fluidosomes(TM)-tobramycin has a highly efficient therapeutic effect."

About Axentis Pharma AG (http://www.axentispharma.com)

Axentis Pharma is a respiratory specialty pharmaceutical company whose core competence is the combination of a fully patented, liposome-based drug delivery system with already established and well-characterized therapeutic agents. The company is using its platform delivery technology, named Fluidosomes(TM) technology, for the development of its lead product, an inhalable liposomal formulation of tobramycin. Axentis Pharma's lead product is designed to treat bacterial infections in the lungs.

About Fluidosomes(TM) technology

Axentis Pharma's Fluidosomes(TM) technology uses biocompatible lipids endogenous to the lung that are formulated into small liposomes. This nanocapsule platform offers wide-ranging potential for unmet medical needs, including chronic respiratory infections of the lung. In the case of Fluidosomes(TM)-tobramycin, the interaction between tobramycin and the microbial cell is triggered when the liposomes undergo a fusion process with the outer membrane of the bacterial cell wall. Tobramycin then penetrates into 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 board@axentispharma.com
    Whttp://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 contact@prd.at
    Whttp://www.prd.at


'/>"/>
SOURCE Axentis Pharma AG
Copyright©2009 PR Newswire.
All rights reserved

Related medicine technology :

1. Promising Cystic Fibrosis Compound on Track for Development
2. Symbollon Pharmaceuticals, Inc. Evaluates the Clinical Effects of IoGen(TM) on Fibrocystic Breast Disease
3. Pediatric Pulmonary Expert Comments on Study of Lung Transplants for Cystic Fibrosis
4. Cystic Fibrosis Foundation Reports Upward Trend for Key Health Outcomes
5. Cystic Fibrosis Foundation Announces Positive Early Results for Phase 2 Clinical Trial of VX-770 - an Oral Compound to Treat CF
6. Lung Transplants in Cystic Fibrosis Patients With Life-Threatening Bacteria Sparks Debate at ISHLT Meeting
7. PTC Therapeutics Announces Data Supporting Cough Frequency as a New Outcome Measure in Evaluating Treatments for Cystic Fibrosis
8. PTC Therapeutics Announces Data from Additional Clinical Studies of PTC124 in Cystic Fibrosis Confirming Activity
9. Transave Announces Positive Phase II Results for Once-Daily Arikace(TM) in the Treatment of Cystic Fibrosis Patients Who Have Pseudomonas Lung Infections
10. Mpex Initiates a Phase 2 Clinical Trial of Aerosol MP-376 in the Treatment of Chronic Bacterial Infections in Cystic Fibrosis Patients
11. The Ocean Becomes a Pipeline To A Cure for Cystic Fibrosis
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/25/2017)...  EpiVax, Inc., a leader in the fields ... announced the launch of EpiVax Oncology Inc., a ... cancer vaccines. EpiVax has provided $500,000 in seed ... technologies to the new precision immunotherapy venture. Gad ... as Chief Executive Officer. Gad brings over 25 ...
(Date:9/19/2017)... Ky. , Sept. 19, 2017   ZirMed Inc ... predictive analytics, today announced that it has been ranked #1 ... Black Book™ Rankings 2017 User Survey. ZirMed was ... solution for large hospitals and medical centers over 200 beds ... Black Book,s healthcare technology user survey history. ...
(Date:9/12/2017)... -- Consumer reviews on the independent review site Consumer Affairs ... for hearing aids, ranking it higher than Miracle Ear ™, ... ... Hearing Aids ... store that provides high performance, state-of-the-art, German-engineered hearing aids directly to ...
Breaking Medicine Technology:
(Date:10/13/2017)... (PRWEB) , ... October 13, 2017 , ... ... agency serving communities in the greater Dallas, Miami, and Raleigh regions, is organizing ... fighting to overcome a rare and deadly chromosome abnormality. , After struggling since ...
(Date:10/13/2017)... ... October 13, 2017 , ... The International Association ... standards of excellence for the field of eating disorders, announces the opening of ... 2018 in Orlando, Florida at the Omni Resort at ChampionsGate. , ...
(Date:10/13/2017)... ... October 13, 2017 , ... Many families have long-term insurance that ... insurance companies have a waiver for care if the client has a cognitive impairment ... family pays for care, is often waived, so the benefits from their insurance start ...
(Date:10/13/2017)... ... October 13, 2017 , ... Talented host, actor Rob Lowe, ... in a new episode of "Success Files," which is an award-winning educational program ... investigates each subject in-depth with passion and integrity. , Sciatica occurs when the ...
(Date:10/12/2017)... CA (PRWEB) , ... October 12, 2017 , ... ... Cheng, are now treating sleep apnea using cutting-edge Oventus O2Vent technology. ... a serious sleep disorder characterized by frequent cessation in breathing. Oral appliances can ...
Breaking Medicine News(10 mins):