Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) In Patients with Nonsense Mutation Dystrophinopathy

Next Previous
X
Search
Trial Profile

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) In Patients with Nonsense Mutation Dystrophinopathy

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 01 Feb 2024

Price : $35 *
  • Adis is an information provider.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
  • A link to download a PDF version of the trial profile will be included in your email receipt.

At a glance

  • Drugs Ataluren (Primary) ; Deflazacort; Prednisolone; Prednisone
  • Indications Duchenne muscular dystrophy; Muscular dystrophies
  • Focus Registrational; Therapeutic Use
  • Acronyms ACT DMD
  • Sponsors PTC Therapeutics

    • Most Recent Events

    • 25 Jan 2024 According to a PTC Therapeutics, Inc. media release, today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarn (ataluren), which will result in the withdrawal of a therapy for patients in Europe. Per European regulations, the European Commission has 67 days to adopt the opinion.
    • 15 Sep 2023 According to a European Medicines Agency media release, CHMP has recommended not renewing the marketing authorisation for Translarna following the full re-evaluation of the benefits and risks of the medicine during the renewal of its marketing authorization. The committee concluded that Translarna's benefit-risk balance is negative. The review considered results from studies, data from a patient registry and the views of consulted experts in neurology and patient representatives.
    • 15 Sep 2023 According to a PTC Therapeutics media release, company announced today that the Committee for Medicinal Products for Human Use of the European Medicines Agency has given a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy on the basis of Study 041, Studies 007 and 020. Company plans to submit a request for re-examination per EMA guidelines.

You need to be a logged in or subscribed to view this content Request demo

If your organization or you do not have a subscription, try one of the following:
  • Contacting your organization’s admin about adding this content to your AdisInsight subscription
  • Buying a PDF version of any individual profile
  • Request a free trial
If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top