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Frequently Asked Questions About Ataluren

PTC Therapeutics Inc. is developing an oral compound designed to treat the underlying cause of cystic fibrosis — a faulty gene and its protein product, called CFTR.

The potential drug, called ataluren (formerly known as PTC124), aims to treat people with CF who have what is known as a nonsense mutation. Nonsense mutations interrupt the production of the CFTR protein and cause it to be too short and not function normally.

Results from a large, international Phase 3 clinical trial of ataluren showed that participants who received the drug had positive trends in lung function and the rate of pulmonary exacerbations, compared with those who received a placebo. 

What is ataluren?

Ataluren (formerly known as PTC124) is a potential new drug for cystic fibrosis that targets a particular type of mutation known as a nonsense mutation.

Nonsense mutations interrupt the production of the CFTR protein, the key protein associated with CF. These mutations cause the protein to be too short and not function normally. About 10 percent of people with CF have nonsense mutations.

For patients with nonsense mutations of CF, ataluren has the potential to address the underlying cause of the disease by allowing a full-length and functional CFTR protein to be made.

Additional information about ataluren is available at People with CF and their families may also contact PTC Therapeutics directly at 1-866-282-5873 or

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What were the results from the Phase 3 clinical trial of ataluren in people with CF?

In June 2012, PTC Therapeutics released results from the Phase 3 study of ataluren in people ages 6 and older with nonsense mutations of CF.

The results showed that participants who received ataluren over the 48 weeks of the study had a lower decline in lung function and a lower rate of pulmonary exacerbations, compared with those who took a placebo (an inactive medication or sugar pill).

Lung function was measured by FEV1 (forced expiratory volume in 1 second). A pulmonary exacerbation is a sudden worsening of symptoms of CF accompanied by a steep decline in lung function.

Safety results from the trial showed that ataluren was generally well tolerated, and that the overall incidence of adverse events (unintended or unwanted events) was similar in people who took ataluren and those on placebo.

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What was the design of the Phase 3 trial?

The study enrolled 238 people ages 6 and older with at least one copy of a nonsense mutation of CF. The trial was conducted at research centers in North America, Europe and Israel and lasted 48 weeks.

Each participant was randomly assigned to one of two treatment groups. One treatment group received ataluren and the other received a placebo. Both groups received the assigned medication three times per day — at morning, midday and evening.

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Is there any way to get ataluren outside of a clinical trial?

Ataluren is an investigational drug that has not yet been approved for use by the FDA. Because ataluren is still in development, it cannot be legally purchased for use by a patient.

Participants who completed the Phase 3 clinical trial of ataluren were eligible to receive ataluren in an ongoing extension study.

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How safe are CF clinical trials?

Nothing is more important than safety in developing new CF treatments. There are four layers of protection in every CF clinical trial. Each trial must be determined as safe and appropriate for patients by the:

  • CF Foundation;
  • FDA;
  • Participating hospital or university’s Institutional Review Board (IRB); and
  • Data Safety Monitoring Board (DSMB). The DSMB is an independent committee of experts in CF care that checks information on ongoing trials, watching for possible problems or unwanted side effects.

The CF Foundation is the only voluntary health organization to organize a DSMB whose members are experts in CF and completely independent and not involved in any way with the trial or its participants. In this way, the CF Foundation does its best to keep participants safe throughout the clinical trial.

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How is my safety protected broadly if I participate in a clinical trial?

The U.S. government has strict guidelines and safeguards to help protect people who choose to participate in clinical research. Every clinical trial in the United States must be approved and monitored by an institutional review board (IRB). This is to keep risks as low as possible and ensure that the risks are worth any potential benefits.

The IRB is usually made up of doctors and the general public. They look at the trial’s protocol (a clear and detailed plan of the experiment) to make sure that participants’ rights are protected and the trial does not cause them unnecessary risk.

In addition, an independent committee of experts in CF care, the Data Safety Monitoring Board (DSMB), examines data from clinical trials and determines if there are safety issues that need to be brought to the attention of the IRB or the sponsors of a clinical trial.

The FDA also must approve all clinical trial protocols and make sure all of their procedures are being followed as the trial goes on.

In the United States, anyone participating in a clinical trial must sign an Informed Consent Form. This form explains the trial in full, including the risks, and a research team member will explain the trial and the consent form to individuals before they sign it.

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Where can I find more information about clinical trials in general?

To learn more about cystic fibrosis clinical trials, click here.

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Updated 6/8/2012

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