Genzyme’s Pompe’s
Treatment Looks Good
So Far For Infantile-Onset Disease
Genzyme, a Cambridge, Mass., biotechnology company, announced
April 26 that an interim analysis of a trial of its experimental
compound Myozyme is “extremely encouraging”
in the treatment of infantile-onset Pompe’s disease, also
known as acid maltase deficiency.
Pompe’s disease is a metabolic muscle disorder that results
from lack of the enzyme acid maltase. The infantile-onset form
of the disease has severe effects on the cardiac and respiratory
muscles and usually leads to death early in childhood.
Genzyme, with supplemental support from MDA, has been testing
Myozyme, a laboratory-engineered compound to replace the missing
acid maltase enzyme, in 18 babies with infantile-onset Pompe’s.
The babies’ disease courses were compared to those of
babies with Pompe’s not treated with Myozyme in the past
(a “historical cohort”). The company says it wanted
to avoid having one group receive Myozyme and the other group
simultaneously remain untreated, as is usual in clinical trials.
The babies in this trial began receiving Myozyme by 6 months
of age. By 12 months of age, 89 percent of those treated with
Myozyme (16 of the 18) were alive and free of invasive ventilator
support, compared with 17 percent in the historical cohort at
12 months.
The interim analysis, reported in an April 26 press release
from Genzyme, also showed that:
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All patients treated with Myozyme showed a reversal in
cardiomyopathy, a condition in which the heart muscle becomes
enlarged and heart function is impaired.
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When the Alberta Infant Motor Scale was used as a measurement,
72 percent of those treated with Myozyme showed gains in motor
(movement) development.
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All patients evaluated showed gains in cognition (thinking),
language, and personal and social skills compared to when
the study began.
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Antibodies (immune-system proteins) to Myozyme developed
in 83 percent of treated patients, and reactions to the Myozyme
infusions occurred in 44 percent.
Genzyme’s press release also notes that the company plans
to submit a biologics license application for Myozyme as a treatment
for Pompe’s disease to the U.S. Food and Drug Administration
(FDA) this year. The company submitted a similar application
to the European Medicines Agency in December.
In addition to supplemental funds for Genzyme’s Myozyme
program, MDA has provided support to Yuan-Tsong Chen at Duke
University, whose work in the 1990s laid the scientific foundation
for Myozyme’s development.
Genzyme is also studying the natural course of late-onset Pompe’s
disease and will soon begin a trial of Myozyme in late-onset
patients.
For information about the Myozyme program, contact Genzyme’s
Medical Information Department at (800) 745-4447, (617) 768-9000,
or medinfo@genzyme.com.
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