The experimental Duchenne muscular dystrophy (DMD) drug ataluren (which has been given the brand name Translarna) has received encouragement from the European Medicines Agency (EMA) for conditional approval in European Union countries.
Ataluren, an oral medication in development by PTC Therapeutics of South Plainfield, N.J., with significant support from MDA, is a stop codon read-through drug, designed to coax muscle cells to ignore ("read through") erroneous stop signals in the genetic instructions for the dystrophin protein and produce fully functional dystrophin.
Dystrophin is missing from muscle cells in DMD patients and diminished in those with the related disorder, Becker muscular dystrophy (BMD).
An erroneous, or "premature," stop signal in the dystrophin gene is thought to cause about 13 percent of DMD cases and may in some instances lead to BMD. Premature stop signals (codons) are also known as "nonsense" mutations.
Positive opinion on conditional approval application
Conditional approval is an EMA designation, roughly equivalent to accelerated approval designation from the U.S. Food and Drug Administration (FDA). Conditional approval status is based on the agency's assessment of a positive benefit-to-risk ratio in the available data, even though the data are not yet considered complete. The drug's developer is required to conduct further studies, but in the meantime, patients can have access to the experimental medication.
A final decision on whether ataluren will receive conditional approval designation is expected within three months.
PTC announced the encouraging news, expressed as a "positive opinion" by the EMA toward possible conditional approval for ataluren, in a May 23, 2014, press release.
"We are very pleased with the outcome of the CHMP [Committee for Medicine Products for Human Use of the EMA] review of Translarna's marketing authorization application and the level of engagement we experienced with CHMP members throughout the review process," said Robert Spiegel, chief medical officer at PTC. "We are grateful to the patients, families, advocacy groups and physicians who have supported PTC Therapeutics through many years of research and development of Translarna. It is important to note that this journey continues through the completion of our phase 3 Translarna confirmatory trial … which is a high priority for PTC and the DMD community."
Phase 3 trial of ataluren (Translarna) remains open
A 220-person, phase 3 trial of ataluren remains open to new participants in the U.S. and several other countries. Participants must be 7-16 years old, have DMD or BMD due to a premature stop codon (nonsense mutation), be able to complete a six-minute walking test, and meet many other criteria.
To participate in the phase 3 trial
For details, see phase 3 trial of ataluren (Translarna), where specific contact information for each location is provided. You also can contact Diane Goetz at PTC Therapeutics (866) 282-5873 or firstname.lastname@example.org.