European Agency to Consider Conditional Approval of Ataluren for DMD

Update (Dec. 6, 2012): This story has been updated with additional information about next steps for ataluren.

New Jersey biopharmaceutical company PTC Therapeutics has announced that the European Medicines Agency (EMA) has validated a "marketing authorization application" (MAA) seeking conditional approval for ataluren, an investigational new drug for the treatment of Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by nonsense mutations (also known as premature stop codons).

According to PTC's Dec. 6, 2012, press release, validation of the MAA confirms that the submission is complete and begins the European agency’s review of the application for conditional approval for ataluren.

The press release says the following about conditional approval by the EMA:

"Conditional approval is granted based on a positive benefit/risk ratio in the available data which, while not yet comprehensive, indicate that the public health benefits of immediate availability of a medicine outweigh its risks. The company is given obligations to fulfill, such as the performance of further studies. The approval is renewed on a yearly basis until all obligations have been fulfilled, and is then converted from a conditional approval into a full approval. Conditional approvals can only be granted for medicines that satisfy an unmet medical need, meaning the medicine is intended to be used for a disease or condition for which no treatment is readily available, and it is therefore important that patients have early access to the medicine concerned."

About ataluren

PTC has been developing ataluren, with initial support from MDA, for people with DMD or BMD due to nonsense mutations in the dystrophin gene.

Nonsense mutations — also known as premature stop codon mutations— cause cells to stop synthesizing a protein from genetic instructions before the process is complete, resulting in production of a short, nonfunctional protein. These mutations are believed to be the underlying cause of DMD or BMD in about 10 to 15 percent of people with these dystrophies, both of which result from a lack of dystrophin in muscle cells.

Ataluren is designed to coax cells to ignore, or "read through," nonsense mutations and synthesize a functional dystrophin protein. The drug is sometimes referred to as a "stop codon read-through" compound.

A 48-week, 174-participant trial suggested a benefit for those on a lower dose of ataluren with respect to the distance walked in six minutes, while participants on high-dose ataluren or a placebo showed no benefit.

In addition to the United States, PTC is conducting clinical trials of ataluren in Australia, Canada, Europe and Israel.

Next steps

In an email to the DMD/BMD community on Dec. 6, PTC CEO Stuart Peltz outlined the company’s next steps toward obtaining approval for ataluren in the U.S. and European Union:

  • PTC is moving forward not only toward European Union approval for ataluren but also toward U.S. regulatory approval.
  • Based on conversations with the U.S. Food and Drug Administration (FDA) and the EMA, PTC plans to enroll participants in a phase 3 confirmatory trial of ataluren as early as the first quarter of 2013.
  • PTC hopes the results of the phase 3 study will serve as the basis for full approval of ataluren in the U.S. and the European Union.
  • PTC expects additional information about the phase 3 trial to be posted on the PTC website and on ClinicalTrials.gov in January 2013.

In his letter, Peltz also said:

"As you know, the path to approval for a treatment in a disorder that has never had a drug approval is not easy or straightforward, but our corporate vision and sense of urgency have never wavered — even when faced with challenges. Instead, our energy and enthusiasm to advance ataluren as rapidly as possible continues to grow, and is reinforced by our robust foundation of preclinical and clinical data."

More information

For background on ataluren (formerly known as PTC124), see:

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