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FDA Grants Orphan Drug Designation for PTC124 for Treatment of Cystic Fibrosis

SOUTH PLAINFIELD, NJ -- December 8, 2004 -- 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 United States Food and Drug Administration (FDA) has granted orphan drug designation to PTC124 for the treatment of cystic fibrosis due to a nonsense mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. Of the 30,000 patients with cystic fibrosis in the United States, it is estimated that 10% are afflicted with the disease due to a nonsense mutation. PTC124 is a novel small-molecule drug currently being evaluated in Phase 1 clinical trials involving healthy volunteers.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to promote the development of products that may offer therapeutic benefits for diseases with a prevalence of fewer than 200,000 individuals per year in the United States. Orphan drug designation provides opportunities for grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States, if granted FDA approval.

"Current treatments for cystic fibrosis consist primarily of management of symptoms, such as the use of antibiotics to combat lung infections," said Langdon Miller, MD, Chief Medical Officer of PTC Therapeutics. "Development of PTC124 provides a potential new option for an underserved patient population because PTC124 has the potential to go beyond palliative care by addressing the underlying cause of this disorder."

Preliminary results from Phase 1 studies have confirmed that PTC124 is orally bioavailable and is well tolerated. Final results from the Phase 1 trials are expected in early 2005. Pending FDA concurrence, PTC expects to advance PTC124 into Phase 2 studies in patients with nonsense-mutation- mediated cystic fibrosis in the first half of 2005. A Phase 2 program in Duchenne muscular dystrophy is also planned for the first half of 2005.

"Orphan drug designation is an important milestone in the development of PTC124, and an achievement with multiple benefits including the opportunity of added mechanisms of funding and communication with the FDA," said Stuart Peltz, Ph.D., President and CEO of PTC Therapeutics.

About PTC124

PTC124 represents a first-in-class, orally delivered investigational new drug for the treatment of genetic disorders due to nonsense mutations. Genetic disorders occur as a consequence of mutations in an individual's DNA. 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 represents a unique opportunity to use a single small-molecule drug to address chronic and life- threatening diseases of high unmet medical need. In addition to cystic fibrosis and Duchenne muscular dystrophy, other potential indications under consideration for PTC124 include hemophilia, neurofibromatosis, retinitis pigmentosa, bullous skin diseases, and lysosomal storage diseases.

About Cystic Fibrosis

Cystic fibrosis is a life-threatening, genetic disease affecting approximately 30,000 people in the United States. A defect in the CFTR gene causes the body to produce abnormally thick, sticky mucus that leads to chronic lung infections and impairs digestion. It is estimated that 10% of the cases of cystic fibrosis are due to a nonsense mutation. More information regarding cystic fibrosis is available through the Cystic Fibrosis Foundation (http://www.cff.org).

SOURCE: PTC Therapeutics, Inc.

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