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BioMarin Announces FDA Approval For Kuvan
BioMarin
Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced that the U.S.
Food and Drug Administration (FDA) has granted marketing approval for
Kuvan(TM) (sapropterin dihydrochloride) Tablets, the first specific drug
therapy approved for the treatment of phenylketonuria (PKU). Shipments to
the distribution channel will commence tomorrow, and BioMarin will begin
promotion of Kuvan immediately.
"The approval of Kuvan represents an important milestone for PKU
patients and their families and also for BioMarin. We are extremely pleased
to bring this promising treatment option to market in just a little over
three years since the IND filing, and we are now ready for an immediate
launch," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.
"We would like to thank all the patients, their families and physicians,
our corporate partners, the FDA, and BioMarin employees for their hard work
and dedication in making Kuvan a reality."
"In clinical trials, Kuvan has been shown to help control blood Phe
levels in PKU patients, and I am thrilled that this new therapy is now
commercially available to the PKU community," stated Dr. Barbara Burton,
Professor of Pediatrics, Northwestern University Feinberg School of
Medicine; Director, PKU Clinic at Children's Memorial Hospital; and
Clinical Investigator in the Kuvan Phase 2 and Phase 3 trials. "With Kuvan
now approved, physicians and patients have, for the first time, a drug
therapy option to manage the disease."
Kuvan is indicated to reduce blood phenylalanine (Phe) levels in
patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4)
responsive PKU and is to be used in conjunction with a Phe-restricted diet.
To determine if there is a response to Kuvan, the recommended starting dose
of Kuvan is 10 mg/kg/day taken once daily for up to a month. If there is no
response, the drug dose may be increased to 20 mg/kg/day for up to a month.
The dose may be adjusted within a range of 5 to 20 mg/kg/day in patients
who respond to Kuvan. Kuvan is developed in partnership with Merck Serono,
a division of Merck KGaA, Darmstadt, Germany.
Clinical Trial Study Results
The efficacy and safety of Kuvan were evaluated in four clinical
studies in patients with PKU.
Study 1 -- A multicenter, open-label, uncontrolled clinical trial of
489 patients with PKU, ages 8 to 48 years (mean 22 years), who had baseline
blood Phe levels greater than or equal to 450 umol/L and who were not on
Phe- restricted diets. All patients received treatment with Kuvan 10
mg/kg/day for 8 days. Response to Kuvan treatment was defined as a greater
than or equal to 30% decrease in blood Phe from baseline. Results: At Day
8, 96 patients (20%) were identified as responders.
Study 2 -- A multicenter, double-blind, placebo-controlled study of 88
patients with PKU who responded to Kuvan in Study 1. After a washout period
from Study 1, patients were randomized equally to either Kuvan 10 mg/kg/day
(N=41) or placebo (N=47) for 6 weeks. Efficacy was assessed by the mean
change in blood Phe level from baseline to Week 6 in the Kuvan-treated
group as compared to the mean change in the placebo group. Results: At
baseline, the mean (+/-SD) blood Phe level was 843 (+/-300) umol/L in the
Kuvan-treated group and 888 (+/-323) umol/L in the placebo group. At Week
6, the Kuvan-treated group had a mean (+/-SD) blood Phe level of 607
(+/-377) umol/L, and the placebo group had a mean blood Phe level of 891
(+/-348) umol/L. At Week 6, the Kuvan- and placebo-treated groups had mean
changes in blood Phe level of -239 and 6 umol/L, respectively (mean percent
changes of -29% (+/-32) and 3% (+/-33), respectively). The difference
between the groups was statistically significant (p < 0.001). Change in
blood Phe was noted in the Kuvan-treated group at Week 1 and sustained
through Week 6.
Study 3 -- A multicenter, open-label, extension study in which 80
patients who responded to Kuvan treatment in Study 1 and completed Study 2
underwent 6 weeks of forced dose-titration with 3 different doses of Kuvan.
Treatments consisted of 3 consecutive 2-week courses of Kuvan at doses of
5, then 20, and then 10 mg/kg/day. Blood Phe level was monitored after 2
weeks of treatment at each dose level. Results: At baseline, mean (+/-SD)
blood Phe was 844 (+/-398) umol/L. At the end of treatment with 5, 10, and
20 mg/kg/day, mean (+/-SD) blood Phe levels were 744 (+/-384) umol/L, 640
(+/-382) umol/L, and 581 (+/-399) umol/L, respectively.
Study 4 -- A multicenter study of 90 children with PKU, ages 4 to 12
years, who were on Phe-restricted diets and who had blood Phe levels less
than or equal to 480 umol/L at screening. All patients were treated with
open-label Kuvan 20 mg/kg/day for 8 days. Response to Kuvan was defined as
a greater than or equal to 30% decrease in blood Phe from baseline at Day
8. Results: At Day 8, 50 patients (56%) had a greater than or equal to 30%
decrease in blood Phe.
Post-marketing commitments include a PKU registry program, a 2-year
extension study for pivotal study patients (ending in mid-2008 for U.S.
patients), a single-dose QT cardiovascular study in healthy volunteers, and
a 7-year open-label clinical study in an estimated 50 PKU patients less
than or equal to 8 years of age. The latter study will verify that control
of Phe levels with Kuvan provides a similar benefit on intellectual
function as expected with dietary Phe restriction. It will also provide
requested safety, efficacy, and pharmacokinetic data in PKU patients less
than or equal to 4 years of age.
BioMarin will offer support to PKU patients through its BioMarin
Patient and Physician Support (BPPS) program. BPPS currently provides
support for MPS VI patients treated with Naglazyme, and a similar support
program will be available for PKU patients.
About Kuvan
Kuvan(TM) (sapropterin dihydrochloride) Tablets is indicated to reduce
blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia
(HPA) due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU).
Kuvan is to be used in conjunction with a Phe-restricted diet.
The active ingredient in Kuvan, 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. BioMarin and Merck Serono estimate that Kuvan
could be a potential treatment option for approximately 30 percent to 50
percent of the estimated 50,000 identified PKU patients in the developed
world.
Kuvan has received orphan drug designation from both the U.S. Food and
Drug Administration (FDA) and the European Medicines Agency (EMEA). Kuvan
has received seven years of market exclusivity in the United States. In
November 2007, Merck Serono submitted a Marketing Authorization Application
(MAA) to the EMEA for sapropterin dihydrochloride as an oral treatment for
patients suffering from HPA due to PKU or BH4 deficiency. If approved in
the EU, it will receive 10 years of market exclusivity for this indication.
Important Safety Information
Prolonged exposure to elevated blood Phe levels in PKU patients can
result in severe neurologic damage. The initiation of Kuvan therapy does
not eliminate the need for careful monitoring of blood Phe levels and
ongoing dietary management.
Some patients receiving Kuvan can experience significant drops in blood
Phe levels. Patients should be monitored closely to ensure that blood Phe
levels do not fall too low.
Not all patients with PKU respond to treatment with Kuvan. Response to
treatment can only be determined by a therapeutic trial of Kuvan.
Kuvan has not been studied in patients with liver or renal impairment.
Patients who have these conditions should be carefully monitored when
receiving Kuvan. Caution should be used with the administration of Kuvan to
patients who are receiving levodopa and drugs that affect nitric
oxide-mediated vasorelaxation or folate metabolism.
The most serious adverse reactions reported during Kuvan administration
(regardless of relationship to treatment) were gastritis, spinal cord
injury, streptococcal infection, testicular carcinoma, and urinary tract
infection. Mild to moderate neutropenia was also noted. The most common
adverse reactions were headache, diarrhea, abdominal pain, upper
respiratory tract infection, pharyngolaryngeal pain, vomiting, and nausea.
To learn more about Kuvan and to access a copy of the full prescribing
information, please visit http://www.Kuvan.com. Information on this website
is not incorporated by reference into this press release.
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 is required for the metabolism of
phenylalanine, 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 becomes toxic to the
brain, resulting in a variety of complications including severe mental
retardation and brain damage, mental illness, seizures, tremors, and
limited cognitive ability. As a result of newborn screening efforts
implemented in the 1960s and early 1970s, virtually all PKU patients under
the age of 40 in developed countries have been diagnosed at birth.
Currently, PKU can only be managed by a Phe-restricted diet, which is
supplemented by nutritional replacement products, like formulas and
specially-manufactured foods; however, the strict diet is difficult for
most patients 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.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for
serious diseases and medical conditions. The company's product portfolio
comprises three approved products and multiple clinical and preclinical
product candidates. Approved products include Naglazyme(R) (galsulfase) for
mucopolysaccharidosis VI (MPS VI), a product wholly developed and
commercialized by BioMarin; Aldurazyme(R) (laronidase) for
mucopolysaccharidosis I (MPS I), a product which BioMarin developed through
a 50/50 joint venture with Genzyme Corporation; and Kuvan(TM) (sapropterin
dihydrochloride) Tablets, a product for the treatment of phenylketonuria
(PKU), developed in partnership with Merck Serono, a division of Merck KGaA
of Darmstadt, Germany. Other product candidates include 6R-BH4 for
cardiovascular indications, which is currently in Phase 2 clinical
development for the treatment of peripheral arterial disease and sickle
cell disease, and PEG-PAL (PEGylated recombinant phenylalanine ammonia
lyase) for the treatment of PKU. For additional information, please visit
http://www.BMRN.com. Information on BioMarin's website is not incorporated
by reference into this press release.
Forward-Looking Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including, without
limitation, statements about: the use of its product Kuvan; the potential
market for Kuvan; expectations regarding filings with regulatory agencies;
and the development of 6R-BH4 for other indications. These forward-looking
statements are predictions and involve risks and uncertainties such that
actual results may differ materially from these statements. These risks and
uncertainties include, among others: the practices of physicians in using
Kuvan; the actual response in patients using Kuvan in commercial
distribution; the content and timing of decisions by the U.S. Food and Drug
Administration, the European Medicines Agency and other regulatory
authorities concerning Kuvan; results and timing of current and planned
clinical trials of 6R-BH4 for other indications; and those factors detailed
in BioMarin's filings with the Securities and Exchange Commission,
including, without limitation, the factors contained under the caption
"Risk Factors" in BioMarin's 2006 Annual Report on Form 10-K, as amended,
and the factors contained in BioMarin's reports on Form 8-K. Stockholders
are urged not to place undue reliance on forward-looking statements, which
speak only as of the date hereof. BioMarin is under no obligation, and
expressly disclaims any obligation to update or alter any forward-looking
statement, whether as a result of new information, future events or
otherwise.
BioMarin Pharmaceutical Inc.
http://www.bmrn.com
View drug information on Aldurazyme; Kuvan; Naglazyme.
BioMarin Announces FDA aprobare pentru Kuvan - BioMarin Announces FDA Approval For Kuvan - articole medicale engleza - startsanatate