SOUTH PLAINFIELD, N.J., March 31 /PRNewswire/ -- PTC Therapeutics, Inc.
(PTC), a biopharmaceutical company focused on the discovery, development, and
commercialization of small-molecule drugs targeting post-transcriptional
control mechanisms, today announced that the company has been granted fast
track designation from the United States Food and Drug Administration (FDA)
for the development of its novel drug, PTC124, for the treatment of cystic
fibrosis (CF). In December 2004, PTC124 was granted orphan drug designation
by the FDA for the treatment of cystic fibrosis due to a nonsense mutation in
the cystic fibrosis transmembrane regulator (CFTR) gene.
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
The fast track program is designed to facilitate the development and
expedite regulatory review of investigational drugs that are intended to treat
life-threatening or severely debilitating diseases and that demonstrate the
potential to address unmet medical needs. Development programs receiving fast
track designations typically receive FDA priority review (6-month vs. standard
10-month review).
PTC124 represents a first-in-class, orally delivered investigational new
drug in development for the treatment of genetic disorders due to nonsense
mutations. Nonsense mutations are single-point alterations in the genetic
code that prematurely halt the translation process, producing a shortened,
non-functional protein. PTC124 allows the cellular machinery to bypass the
nonsense mutation and continue the translation process, restoring the
production of full-length, functional proteins. Single and multiple-dose
Phase 1 studies involving healthy volunteers have been recently completed, and
PTC is working with investigators and the FDA to finalize plans for Phase 2
studies in CF and Duchenne muscular dystrophy (DMD).
"We continue to make excellent progress in this program and believe that
PTC124 has the potential to provide a treatment for CF patients who have the
disease due to a nonsense mutation," said Stuart W. Peltz, Ph.D., President
and CEO of PTC. "PTC124 is unique in that it addresses the underlying genetic
cause of the disease, not just the symptoms. We were granted orphan drug
designation in CF and DMD recently, and fast track designation is an important
added component to the development of PTC124 for patients who are in desperate
need of new treatment options."
About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery, development,
and commercialization of small-molecule drugs targeting post-transcriptional
control mechanisms. Post-transcriptional control processes are the sequence
of events in the cell that ultimately regulate the rate and timing of all
protein production. PTC's compounds alter these processes by selectively
modulating how RNA is used to produce proteins. By applying this approach,
PTC has advanced its drug discovery programs rapidly from targets to
preclinical and clinical drug candidates, building a robust pipeline across
genetic disorders, oncology, and infectious diseases.
About PTC124
PTC124 represents a first-in-class, orally delivered investigational new
drug for the treatment of genetic disorders due to nonsense mutations.
Nonsense mutations are single-point alterations in the genetic code that
prematurely halt the translation process, producing a shortened,
non-functional protein. PTC124 allows the cellular machinery to bypass the
nonsense mutation and continue the translation process, restoring the
production of full-length, functional proteins. PTC124 has demonstrated the
ability to restore full-length functional protein in genetic disease models
harboring nonsense mutations. PTC124 represents a unique opportunity to use a
single small-molecule drug to address chronic and life-threatening diseases of
high unmet medical need. It is estimated that 10% of the cases of cystic
fibrosis (CF) and 15% of the cases of Duchenne muscular dystrophy (DMD) are
due to nonsense mutations. PTC has catalogued over 1,800 distinct genetic
disorders where nonsense mutations are the cause of the disease in an
appreciable percentage of patients. In addition to CF and DMD, other
potential indications under consideration for PTC124 include hemophilia,
neurofibromatosis, retinitis pigmentosa, bullous skin diseases, and lysosomal
storage disorders.
About Cystic Fibrosis
Cystic fibrosis (CF) is a life-threatening, genetic disease affecting
approximately 30,000 people in the United States. A defective gene causes the
body to produce abnormally thick, sticky mucus that leads to chronic
lung-infections and impairs digestion. More information regarding CF is
available through the Cystic Fibrosis Foundation ( http://www.cff.org ).
SOURCE PTC Therapeutics, Inc.
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Related links:
http://www.ptcbio.com
http://www.cff.org
Photo Notes:http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
CONTACT:
Jane Baj of PTC Therapeutics, Inc.,
+1-908-222-7000, ext. 167, jbaj@ptcbio.com; or Robert Stanislaro
of Noonan Russo, +1-212-845-4268, robert.stanislaro@eurorscg.com,
for PTC Therapeutics, Inc.
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