Please login to the form below

Not currently logged in

FDA reviewer unimpressed with PTC’s Translarna

Finds the muscular dystrophy treatment's evidence to be 'not persuasive'


PTC Therapeutics’ Duchenne muscular dystrophy drug Translarna is up for an FDA advisory committee meeting on Thursday - but is facing resistance from the agency’s reviewer.

In a damning report, the reviewer suggests that PTC “has presented only the results from numerous post hoc and exploratory analyses that are intended to mitigate two negative clinical trials” and that the overall package submitted by the company is “not persuasive”. Shares in PTC fell sharply after the FDA document was published.

The dossier for Translarna (ataluren) has already been in front of the FDA twice and rejected both times, and analysts from Credit Suisse and JP Morgan both suggested that the chances of it being a case of ‘third time lucky’ for the drug in the US are slim. It is already approved for sale in Europe as well as countries in the Middle East and Latin America and has been launched in more than 25 countries worldwide - with PTC modelling sales of $140m this year. Its conditional EU approval was renewed in January but remaining on the market will depend on the outcome from a long-term trial due in 2021.

The strong terms used by the FDA reviewer begs the question why PTC decided to press ahead with another regulatory filing for the drug in the US, and it may be that the firm was emboldened by the FDA’s approval last year of another DMD therapy from Sarepta - Exondys 51 (eteplirsen) - after Janet Woodcock, head of the agency’s Center for Drug Evaluation and Research, took the unusual step of overruling a decision by an advisory committee.

As recently as the company’s second-quarter results call last month, PTC’s chief executive Stuart Peltz said PTC was “actively preparing for a potential US launch” of the drug, saying its commercial operations were already in gear following the FDA’s approval of Emflaza (deflazacort) for DMD earlier this year.

PTC recently shared additional data from a phase III trial of Translarna showing that the drug slightly slowed disease progression in ambulatory boys with Duchenne muscular dystrophy, but was unable to show a significant difference from placebo.

Article by
Phil Taylor

27th September 2017

From: Regulatory



PMEA Awards 2020

COVID-19 Updates and Daily News

Featured jobs


Add my company

AMICULUM® is an independent global healthcare communications, consulting and learning business with a global team of >220 healthcare communications professionals,...

Latest intelligence

Good design saves lives
Good design and creative thinking are essential if we are to improve on existing problems in new ways, which is why design and creativity within healthcare is vital. Health is...
Why you must understand the pricing of patient recruitment companies
Recruiting a diverse range of patients and engaging with them for your clinical trial isn’t an easy task, which means you might turn to patient recruitment companies, like us, who...
wearable health tech
A cultural shift in clinical research
Research organisations across the board are experimenting with new technologies...