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BioMarin Announces Positive Results From Phase 3 Extension Study Of Phenoptin For PKU
BioMarin
Pharmaceutical Inc. (Nasdaq and SWX: BMRN) today announced positive results
from the pivotal Phase 3 extension study of Phenoptin (sapropterin
hydrochloride), an investigational oral small molecule for the treatment of
patients with phenylketonuria (PKU), who have elevated phenylalanine (Phe)
levels. Results confirm that all pre-specified safety and efficacy
endpoints of the open-label extension study were met. Data demonstrated the
long-term safety and tolerability of Phenoptin as a treatment to control
blood Phe levels across a range of doses in PKU patients.
The 22-week multi-center open-label dose titration Phase 3 extension
study enrolled 80 patients who had previously completed the pivotal Phase 3
clinical trial. Patients in the extension study received Phenoptin doses of
5 mg/kg/day, 10 mg/kg/day, and 20 mg/kg/day for two-week intervals each,
followed by four weeks at 10 mg/kg/day. Patients completing this first
phase of treatment were then treated for an additional 12 weeks with one of
the three doses, depending upon their Phe levels at weeks two and six. A
total of 79 patients completed both stages of treatment.
Key findings from the study:
-- Phenoptin demonstrated an excellent safety and tolerability profile for
all three doses over the 22 weeks of treatment, which was the primary
endpoint of the extension study. There were no withdrawals due to
adverse events related to the study drug during the extension study.
-- Phenoptin provided a dose-dependent reduction in blood Phe levels
relative to baseline, with average Phe level decreases of 100 umol/L
(1.7 mg/dl), 204 umol/L (3.4 mg/dl), and 263 umol/L (4.4 mg/dl) for
patients receiving doses of Phenoptin of 5 mg/kg/day, 10 mg/kg/day, and
20 mg/kg/day, respectively (secondary endpoint).
-- A once daily dose regimen of Phenoptin was sufficient to maintain the
reduction of blood Phe levels throughout a 24 hour period.
-- The incidence and type of adverse events were comparable to that of the
placebo group during the double-blind study and nearly all were mild or
moderate in severity. The four most frequently reported events were
headache, nasopharyngytis, vomiting, and diarrhea.
The ongoing Phase 3 diet study is designed to assess the increase in
Phe tolerance levels in patients taking Phenoptin, and BioMarin remains on
track to announce results from the Phase 3 diet study in the first quarter
of 2007. BioMarin and Serono expect to file the NDA and MAA in the second
and third quarters of 2007, respectively. In May 2005, BioMarin entered
into a strategic partnership with Serono for the development and
commercialization of Phenoptin for the treatment of PKU.
About Phenoptin
Phenoptin is an investigational oral small molecule therapeutic for the
treatment of PKU. The active ingredient in Phenoptin, sapropterin
dihydrochloride, is the synthetic form of 6R-BH4 (tetrahydrobiopterin), a
naturally occurring enzyme cofactor that works in conjunction with
phenylalanine hydroxylase (PAH) to metabolize Phe. Preliminary clinical
data have suggested that Phenoptin has a potential to produce significant
reductions in blood Phe levels in the subset of patients who are BH4-
responsive. BioMarin and Serono estimate that Phenoptin could be a
potential treatment option for approximately 30 percent to 50 percent of
the estimated 50,000 individuals in the developed world who have been
diagnosed with PKU.
Phenoptin received orphan drug designation to treat PKU from both the
U.S. Food and Drug Administration (FDA) and European Medicines Agency
(EMEA). If Phenoptin becomes the first drug therapy approved for the
treatment of PKU, Phenoptin would receive seven years of market exclusivity
in the United States and 10 years in the European Union for this
indication. Additionally, the FDA has granted Phenoptin Fast Track
designation, which is designed to facilitate the development and expedite
the review of new drugs that are intended to treat serious or
life-threatening conditions and that demonstrate the potential to address
unmet medical needs.
About PKU
PKU, a genetic disorder affecting approximately 50,000 diagnosed
patients in the developed world, is caused by a deficiency of the enzyme
phenylalanine hydroxylase (PAH). PAH is required for the metabolism of
phenylalanine (Phe), an essential amino acid found in most
protein-containing foods. If the active enzyme is not present in sufficient
quantities, Phe accumulates to abnormally high levels in the blood and
brain, resulting in a variety of complications including severe mental
retardation and brain damage, mental illness, seizures and tremors, and
cognitive problems. As a result of global newborn screening efforts
implemented in the 1960s and early 1970s, virtually all PKU patients in
developed countries have been diagnosed at birth. The only treatment
currently available for PKU patients is a highly restrictive and expensive
medical food diet that most patients fail to adhere to the extent needed
for achieving adequate control of blood Phe levels. To learn more about
PKU, please visit http://www.PKU.com. Information on this website is not
incorporated by reference into this press release.
Positive Results from Phase 3 Clinical Study of Phenoptin for PKU
Positive results of a Phase 3, double-blind, placebo-controlled
clinical study of Phenoptin (sapropterin dihydrochloride), an
investigational oral small molecule for the treatment of phenylketonuria
(PKU) were reported on March 15, 2006. Results confirmed that all
pre-specified primary and secondary endpoints were met and data
demonstrated a statistically significant reduction at six weeks in blood
phenylalanine (Phe) levels (p
BioMarin anunþã rezultate pozitive de la faza 3 de studiu de extensie Phenoptin pentru PKU - BioMarin Announces Positive Results From Phase 3 Extension Study Of Phenoptin For PKU - articole medicale engleza - startsanatate