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news.The US Food and Drug Administration (FDA) has rejected PTC Therapeutics' new drug application (NDA) for Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD).
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In its refuse to file letter, the agency stated that PTC’s application was insufficient to permit a substantive review.
The company said it is reviewing its content to identify the relevant steps that are to be followed.
PTC discovered and developed translarna, which is a protein restoration therapy designed to allow the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
The company said a nonsense mutation is an alteration in the genetic code that prematurely stops the synthesis of an essential protein.
The resulting disorder is identified by which protein cannot be expressed in its entirety and is no longer functional, like dystrophin in DMD.
Translarna is licensed in the European Economic Area to treat nmDMD in ambulatory patients 5 years of age and older. It is an investigational new drug in the US.
DMD, which primarily affecting males, is a progressive muscle disorder caused by the lack of functional dystrophin protein.
PTC’s internally discovered pipeline addresses several therapeutic areas such as rare disorders and oncology.
The company discovered all of its compounds currently under development using its technologies. PTC intends to continue the development of the compounds both on its own and via selective collaborations with pharmaceutical and biotechnology firms.