- The Food and Drug Administration has notified PTC Therapeutics of a "tentative scheduling" of the Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the company's Duchenne muscular dystrophy (DMD) drug, Translarna (ataluren).
- Translarna, which has faced a tortuous route to this point, now has a Prescription Drug User Fee Act goal date of October 24, 2017.
- Investors see this as a positive move, shifting share price up to $14.31 at close of market yesterday after a swing of over 9%.
PTC Therapeutics' stock has been up and down like a yo-yo over the last 18 months, and the company is pinning its hopes on the tentatively scheduled advisory committee meeting after a slew of setbacks dating back to its first U.S. submission for approval in January 2016.
Translarna missed primary endpoints in its mid- and late-stage DMD studies, but the company still moved ahead with submissions. The FDA rejected Translarna's initial rolling submission with a "refusal to file" letter for an incomplete application in February 2016, following this up in October 2016 with a denial of the company's appeal. The refusal to file was a knock back for PTC, leading to layoffs of almost a fifth of its workforce.
In its statement at the time of the FDA's appeal denial, PTC said it saw it as "an iterative process and the company anticipates that multiple cycles of appeals to progressively higher levels of the FDA may be required," which isn't perhaps necessarily how the FDA sees the process.
PTC's next step was a rare one. It submitted a filing over protest, which forced the FDA's hand, ensuring a review of the new drug application. The FDA granted standard review and set the October user fee date, which will be just a month after the adcom meeting.
Translarna isn't having an easy route to approval in DMD in Europe either. It gained conditional approval in 2014. In November 2016, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended renewing the conditional marketing approval in late 2016. In January 2017, Translarna received an annual renewal with a request to carry out an additional Phase 3 trial in around 250 DMD patients.
PTC isn't having much luck with the drug elsewhere. PTC admitted defeat on cystic fibrosis when Translarna failed to meet both primary and secondary endpoints in a Phase 3 trial, and that indication has now been shelved.
RBC Capital Markets analyst Matthew Eckler said in a note to clients that the FDA's decision to hold the advisory committee meeting is driven by "being careful to check every box during review" because of the media, political and patient advocacy attention focused on the drug, rather than any signs that it will produce a positive decision.
"We continue to ascribe value to ataluren based solely on its ex-US commercial prospects, where we model peak sales of around $250 million. However, if the September panel goes ataluren’s way, and the drug makes it onto the U.S. market, this could potentially add another $500 million to our revenue estimates, representing a significant source of upside to our valuation," he wrote.