"We are very excited to be advancing our second indication, which represents another milestone in realizing the full potential for Translarna." said
"Nonsense mutation cystic fibrosis is a serious form of cystic fibrosis and is very challenging to treat. We are pleased that there may be a treatment option for people with nmCF in the near future," said
The regulatory application for Translarna for nmCF is based on previously announced analyses from the company's prior completed Phase 3 double-blind, placebo-controlled study comparing Translarna to placebo in nmCF patients. PTC is conducting an additional randomized, double-blind, placebo-controlled Phase 3 study of Translarna in nmCF patients and expects enrollment to be completed by the end of this year, with top-line data expected by the end of 2016.
About Translarna™ (ataluren)
Translarna, discovered and developed by
Translarna has the potential to benefit patients across the vast array of genetic disorders caused by a nonsense mutation. On average, 11% of every monogenic disorder is caused by a nonsense mutation. PTC's strategy is to rapidly expand the clinical development of Translarna across multiple genetic disorders to deliver on the company commitment to address rare and neglected disorders.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare disabling and life-threatening genetic disorder resulting from mutations that cause the lack or dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, a chloride ion transport channel which results in thick mucus secretions in the lung, pancreas and other organs. In patients who have cystic fibrosis due to a nonsense mutation, an interruption in the genetic code prematurely halts the synthesis of CFTR, causing the protein to be short and non-functioning. Nonsense mutations are categorized as Class I mutations that result in little or no production of the CFTR protein, which makes people with these mutations very difficult to treat. CF patients with Class I mutations typically experience more severe disease symptoms than those with other genotypes, including a shorter life span, a higher probability of end-stage lung disease, and a higher prevalence of pancreatic insufficiency. Approximately 10% of patients have CF due to a Class I nonsense mutation in at least one allele of the CFTR gene. Available therapies for treatment of lung manifestations of CF, such as inhaled antibiotics do not address the underlying defect. There are no marketed treatments that target the defect associated with CF caused by nonsense mutations.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA, or mRNA, molecule is copied from DNA through the transcription process. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com.
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Forward Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements, other than those of historical fact, contained in this release are forward-looking statements, including statements regarding the future expectations, plans and prospects for PTC; the rate and degree of market acceptance and clinical utility of Translarna; PTC's estimates regarding the potential market opportunity for Translarna, including the size of eligible patient populations and PTC's ability to identify such patients; the timing and conduct of PTC's clinical trial of Translarna for the treatment of nmCF, including statements regarding the timing of initiation, evaluation, enrollment and completion of the trial and the period during which the results of the trial will become available; matters related to our current and planned regulatory filings, including timing for EMA review of our variation submission for nmCF, whether the EMA will grant a positive opinion with respect to such submission, and the timing and determinations of the
PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to the EMA's determinations with respect to our nmCF variation submission ; the initiation, conduct and availability of data from clinical trials and studies; expectations for regulatory approvals; PTC's scientific approach and general development progress; the eligible patient base and commercial potential of Translarna and PTC's other product candidates and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q as well as any updates to these risk factors filed from time to time in PTC's other filings with the
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