Ataluren is the active ingredient of an oral drug candidate developed by PTC Therapeutics. The small-molecule compound is designed to treat cystic fibrosis (CF) patients with nonsense mutations, allowing the cell to produce full functional proteins. In the U.S., the therapy is being tested in a Phase 3 trial with CF patients who have nonsense mutations and are not taking aminoglycosides (an antibiotic).
In genetics, a nonsense mutation is a mutation in a DNA sequence that results in a shorter, unfinished, and mostly nonfunctional protein product.
History of ataluren
According to a PTC press release, Phase 2 trials for ataluren were initiated in 2010. Later that year, pivotal data suggested that the drug had therapeutic potential for patients carrying nonsense mutations because it demonstrated the ability to slow down the disease’s progression.
Phase 2 and Phase 3 studies continued to show positive results for protein restoration, as reported in 2013.
In 2014, atularen received approval from the European Medicines Agency (EMA) to treat patients with Duchenne muscular dystrophy and it was launched under the trademark name Translarna. In the same year, ataluren was designated by the EMA and U.S. Food and Drug Administration (FDA) as an orphan drug for the treatment of both Duchene muscular dystrophy and cystic fibrosis.
Phase 3 results for Duchenne muscular dystrophy were released in 2015, confirming the drug’s ability to slow down the progression of the disease.
In the U.S., a Phase 3 extension trial for patients with CF who are not taking aminoglycosides is currently enrolling participants.
How ataluren works
In healthy people, full-length and functional proteins are produced by mRNA molecules. In people carrying nonsense mutations, stop codes signal the premature end of protein synthesis. It is believed that ataluren interacts with the ribosome of a cell, enabling it to read stop signals and allow the cell to produce complete proteins.
Next steps for ataluren
According to both ClinicalTrials.gov and PTC Therapeutics sites, a Phase 3 trial for patients with nonsense mutation CF who are not taking aminoglycosides is ongoing. The drug is not yet approved by the FDA and its access in the U.S. is only through clinical trials.
The drug is approved in Europe to treat patients with nonsense mutations in Duchenne muscular dystrophy. Its use for patients with this condition is still awaitng FDA approval.
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