KOSTENDALINGEN
bij
gebruik en toediening
van
MYOZYME
Veelbelovende innovaties en verbeteringen: Amicus, Oxyrane, Biomarin, Gentherapie
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Vergevorderde trials bij Amicus: AT2220 verhoogt de opname van Myozyme aanzienlijk Mark Tarnopolsky, McMaster Univ. showed "A Phase 2 Study to Investigate Drug-Drug Interactions between escalating doses of AT2220 and rhGAA in subjects with pompe Disease". So this is the combination of Amicus' AT2220 and ERT and it has shown to increase active levels of rhGAA in both blood and muscle. There appeared to be a "dose dependent" escalation of rhGAA when combined with increasing AT2220. There will be a follow up clinical Trial to help establish the optimal dosing and confirm these finding. Cross your fingers with this one as it could really help stabilize ERT if it works and so far so good! Pre-klinisch succes bij Oxyrane: 20-voudige verbeterde opname van Pompe-enzym Oxyrane, founded in 2006, develops enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders (LSDs). Oxyrane’s lead product is OXY2098-95 which is currently in pre-clinical testing for Pompe Disease. Pompe disease is a rare disorder where the progressive accumulation of glycogen results in muscle weakness – particularly in the heart and skeletal muscle. In November 2012 Oxyrane announced positive results from a study undertaken in collaboration with a Belgian University showed that LSDs can be treated with modified enzymes such as OXY2098-95. The increased level of targeting shown in the trial resulted in a 20 fold increase in cellular uptake of the enzyme, thus having the potential to significantly improve treatment for patients with LSDs. This trial has laid the foundations for Oxyrane to move forward into clinical development. Oxyrane now hopes to move forward into clinical development in the coming months with a Phase I trial being planned. Oxyrane also has 4 other programmes they are hoping to progress in various LSD indications. Biomarin: Verhoging Enzymopname dankzij GILT
POM
-
001 is a phase I/II open
label dose
finding study of the safety and
tolerability of BMN 701
in patients with late
onset Pompe Disease
that has been open approximate
ly 1 year. Patients
are treated by intravenous
infusion every 2 weeks for up to
24 weeks. Three dose levels of
BMN 701 are being investigated
(5, 10 & 20 mg/kg). The study’s
primary endpoint is to
determine the immune response to
the drug. Secondary en
dpoints include pharmacokinetics,
and preliminary efficacy
assessments. A preliminary
report on the POM
001 study was
presented by Dr. Bruce Barshop (University
of California, San Diego) in
February at the 8
th
Annual WORLD Symposium for
Lysosomal Storage
Disorders. So far, six patients
have been
treated at the 5 & 10 mg/kg dose
levels for up to 24 weeks. The
first subject began dosing at
the 20 mg/kg dose level in
January 2012 after an
independent review board
permitted dose
escalation. UK study sites
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