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Cystic Fibrosis – Liposomal Tobramycin Receives Second Orphan Drug Designation Within Weeks

July 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
    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 contact@prd.at
    W http://www.prd.at

SOURCE Axentis Pharma AG


Source: newswire



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