PTCT's Quest For Approval Of DMD Drug Translarna

(RTTNews) - After a harsh internal review on Tuesday, there was very little hope of PTC Therapeutics' (PTCT) Duchenne muscular dystrophy drug Translarna being recommended for approval by the panel of outside experts convened by the FDA on September 28, 2017.

As expected, yesterday, the FDA panel voted 10-1 that the data provided by the Company was inconclusive to recommend approval of Translarna. The panel also stated that the Company needs to provide more data to prove the drug's efficacy.

Translarna, also known as ataluren, is designed to treat certain nonsense mutation Duchenne muscular dystrophy (nmDMD) patients.

Despite a failed phase III clinical trial of Translarna in DMD patients, the Company went ahead in seeking approval of the drug in January of 2016. But the FDA refused to even review the application then by issuing a Refuse to File Letter last February. And in October 2016, the Company's first appeal of the refuse to file letter was also denied by the Office of Drug Evaluation I of the FDA.

Escalating its appeal to the next supervisory level of the FDA, PTC filed the NDA for Translarna using the FDA's "file over protest regulations", and it was accepted for review in March, 2017.

In June of this year, the regulatory agency notified the Company that a panel of outside experts will be reviewing the Translarna NDA before a final decision is made.

Now that the panel has voted against approval of Translarna, it's now over to the FDA. The FDA usually follows the advice of its advisory panels although it is not obliged to do so.

So, is the writing already on the wall for Translarna or like in the case of Sarepta Therapeutics' DMD drug Exondys 51, will the FDA disagree with the panel's recommendation? We will know that by October 24, 2017 when the FDA's final decision will be announced.

Translarna is approved within the European Union (EU) Member States, Iceland, Liechtenstein, Norway, Israel and South Korea. Ever since its launch in December 2014, Translarna has had substantial year-over-year net sales growth.

Sales of Translarna, which were $34 million in 2015, more than doubled to $81 million in 2016. Looking ahead to full-year 2017, the Company expects Translarna to bring home sales between $120 million and $140 million.

PTCT closed Thursday's trading at $17.46.

Now, let's take a look at some of the biotech stocks that grabbed headlines this week.