SOUTH PLAINFIELD, N.J., Dec. 21, 2010 /PRNewswire/ — PTC
Therapeutics, Inc. announced today that it has completed enrollment
of a Phase 3 clinical trial of ataluren, an investigational new
drug, in patients with nonsense mutation cystic fibrosis
(nmCF).
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The 48-week study is designed to determine whether ataluren can
improve lung function in patients with nmCF. The trial has
enrolled 238 patients at 36 sites in North America, Europe and
Israel. Patients who complete the treatment phase of the
Phase 3 trial are eligible to participate in a 48-week, open-label
extension study, which has begun enrolling patients.
“The enrollment of this trial represents an important step
forward in our efforts to develop disease-modifying treatments that
advance the standard of care in CF and improve quality of life for
CF patients,” stated Michael Konstan, MD, Chairman, Department of
Pediatrics at Rainbow Babies and Children’s Hospital in Cleveland,
Ohio. Dr. Eitan Kerem, Head, Department of Pediatrics and CF
Center, Hadasash University Hospital, Jerusalem, Israel added,
“Despite significant advances in the 21 years since the
identification of the disease-causing gene, cystic fibrosis remains
a debilitating and life-threatening disorder and available
therapies focus only on alleviating symptoms. Ataluren couples a
patient’s genetic diagnosis with a mutation-specific therapeutic
approach designed to address the underlying cause of the
disease.”
Ataluren is a protein restoration therapy designed to enable the
formation of full-length, functional cystic fibrosis transmembrane
regulator (CFTR) protein in patients with cystic fibrosis due to a
nonsense mutation. CFTR is a critical protein lacking in CF
patients. Nonsense mutations are categorized as Class I
mutations that result in little or no production of the CFTR
protein. CF patients with Class I mutations typi
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