Dive Brief:
- PTC Therapeutics faces further regulatory delays in Europe for its Duchenne muscular dystrophy drug, announcing Monday a renewal of Translarna's conditional approval would not be completed by mid-2016.
- Translarna's current conditional authorization will remain valid until the European Commission makes a final decision, PTC said. The drug won conditional approval from the European Medicines Agency in 2014, subject to annual renewal and review.
- Since then, however, PTC Therapeutics was hit with a "refusal to file letter" from U.S. regulators, who deemed the application for approval of Translarna to be incomplete. In its update Monday, PTC said it had appealed to escalate discussions to the next level of management at the Food and Drug Administration as part of the dispute-resolution process. PTC did, however, indicate willingness to prepare an additional clinical trial.
Dive Insight:
Translarna has received a fair share of scrutiny over the clinical data it used to gain conditional approval in Europe in 2014, and today's announcement that even the EMA would require another clinical trial indicates the "conditional" clause in the drug's approval may have run its course.
Although the drug has been in the European market for over a year now, the Food and Drug Administration refused to even consider the drug, arguing the Phase 2b trials and a failed Phase 3 trial were insufficient evidence for conditional approval.
The rejection led to a rough stretch for the company: PTC's stocks plunged by nearly 60% following the FDA announcement, the company proceeded to cut 18% of its staff in a New Jersey plant the next month, and in April the Scottish healthcare system rejected a healthcare licensing deal based on similar data concerns.
It hasn't been all bad news, though: just a few weeks ago, the company reached a Managed Access Agreement with National Health Services England, through which the two parties will monitor the progress of a much larger patient pool.
PTC said European health regulators had agreed to review a draft clinical trial protocol aimed at shoring up the marketing authorization of Translarna. While the company voiced its optimism in winning eventual renewal, a further clinical trial will likely be needed.
In the U.S., however, the company is still exploring the "possibility of conducting an additional clinical trial under accelerated approval."
Investors do not appear to have taken kindly to the announcement—stocks had fallen by up to 6% as of press time.