Ataluren manufacturers

About ataluren

Ataluren manufacturers are limited to the originator, PTC Therapeutics, which marketed it as Translarna. Ataluren is an oral drug designed to let cells read through nonsense mutations and make full-length dystrophin in Duchenne muscular dystrophy (DMD). The EU did not renew its conditional authorisation in March 2025, and PTC withdrew its US application in February 2026. Ataluren remains available only in a few countries and through special access schemes. Licensing opportunities are therefore very limited.

Ataluren key facts

Ataluren manufacturers and licensing history

PTC Therapeutics developed ataluren as the first drug targeting nonsense mutations in DMD. The EU granted conditional approval on 31 July 2014 for ambulatory patients aged 5 and over, later extended to age 2.

Conditional approval required confirmatory data. After reviewing the results, the CHMP concluded that effectiveness had not been confirmed. The European Commission decided not to renew the authorisation on 28 March 2025.

In the US, the FDA refused to file the first application in 2016 and rejected it in 2017. PTC resubmitted in 2024, but withdrew the application on 12 February 2026 after the FDA said the data were unlikely to meet its standard. PTC is supplying US patients already on treatment while existing stock lasts. Ataluren is now available only in a few other countries and through special access schemes.

Ataluren dosage forms and dossier types

Ataluren is supplied as granules for oral suspension in sachets, dosed three times a day by body weight.

Ataluren regulatory and patent status

Ataluren received conditional EU approval on 31 July 2014. On 28 March 2025, the European Commission decided not to renew it, because effectiveness had not been confirmed. In the US, PTC withdrew its resubmitted application on 12 February 2026 after FDA feedback. Ataluren remains registered in some countries outside the EU and US, and some EU countries allow access through national schemes.

Ataluren mechanism of action and therapeutic use

A nonsense mutation creates a premature stop signal in a gene, so the cell stops making the protein too early. Ataluren is designed to let the ribosome read through this stop signal and produce full-length dystrophin. It was used for ambulatory patients with DMD caused by a nonsense mutation, confirmed by genetic testing.

Ataluren licensing and sourcing

Ataluren is available only from PTC Therapeutics, in limited markets. Companies interested in this area usually look at named-patient supply or other DMD therapies. Current offers and contacts are listed among the ataluren manufacturers on Pipelinepharma.

Frequently asked questions about ataluren

Who manufactures ataluren? PTC Therapeutics is the only manufacturer and sold ataluren as Translarna.

Is ataluren still approved in the EU? No. The European Commission decided not to renew Translarna's conditional authorisation on 28 March 2025, because effectiveness had not been confirmed.

Is ataluren approved in the US? No. PTC withdrew its US application on 12 February 2026 after the FDA said the data were unlikely to support approval.

What forms and strengths of ataluren are available? Ataluren was supplied as granules for oral suspension in 125, 250 and 1,000 mg sachets.

Can patients still get ataluren? Ataluren remains available in some countries outside the EU and US and through national special access schemes. In the US, PTC is supplying patients already on treatment while stock lasts.