Two Firms Say Fabry Drugs Succeed in Trials
Last updated: 06 Oct 2000 00:37 GMT (Reuters)
NEW YORK (Reuters) - Transkaryotic Therapies Inc.
TKTX.O and Genzyme General Corp. GENZ.O said on
Thursday their rival experimental treatments for a rare
inherited disorder known as Fabry disease both
proved successful in late-stage clinical trials.
The companies, both based in Cambridge, Mass.,
unveiled their data in Philadelphia at an annual
meeting of the American Society of Human Genetics.
An estimated 2,000 to 4,000 people around the world
suffer from the disease, caused by deficiency of the
enzyme alpha-galactosidase. Without the enzyme,
patients are unable to break down blood fats, which
then collect in blood vessels of the kidney and other
organs and typically cause death by age 40.
The companies are awaiting approval from the U.S.
Food and Drug Administration for enzyme-replacement
drugs meant to reduce pain and alleviate symptoms
by allowing the body to perform its normal role of
keeping blood lipids in balance.
Transkaryotic's (TKT) pivotal Phase II trial of its drug
Replagal involved 26 patients, half of whom received
infusions of the enzyme replacement every two weeks
and half of whom received placebo infusions.
After six months of treatment with Replagal, patients
showed clinically and statistically significant reduction
in pain and stabilization of kidney function and
improvement in kidney pathology, researchers said.
"There also were extensive decreases in accumulation
of globotriaosylceramide (a blood lipid also known as
Gb3) in various organs that were significant," said Dr.
Raphael Schiffmann, principal researcher for trials of
the drug conducted at the National Institutes of
Health.
Genzyme General's drug Fabrazyme was studied in a
Phase III trial involving 58 patients at hospitals in the
United States and Europe. Intravenous dosing was
also every two weeks, with half the group taking
Fabrazyme and half taking placebos.
Genzyme said Fabrazyme was highly successful in its
main trial goal of almost completely clearing blood lipid
GL-3 from blood vessels of the kidney.
"Twenty of the 29 patients treated with Fabrazyme
achieved a score of zero, indicating that their blood
vessels were restored to a near-normal state and
providing strong evidence that the kidney should
function normally as a result of treatment," Genzyme
said in a prepared release.
It said eight of the remaining nine patients who
received Fabrazyme also improved but did not achieve
a score of zero during the first 20 weeks of
treatment. None of the 29 placebo patients achieved
a score of zero, it added. |
