PTC Therapeutics’ Duchenne muscular dystrophy drug Translarna (ataluren) failed a pivotal Phase III trial and recently was spurned by the FDA, which refused to even consider the biotech’s ($PTCT) application after regulators concluded that the company couldn’t make a case on efficacy.
But it’s now good enough for NICE and U.K. patients.
The U.K.’s drug pricing watchdog initially rejected the drug a year ago, but today--after boys with Duchenne MD and their families mounted a high-profile lobbying effort--the regulatory agency announced that the drug will be recommended after PTC agreed to an undisclosed discount on the $300,000 retail price for the drug. PTC and the U.K.'s National Health Service are now completing a final agreement for providing the drug, which is expected to be wrapped in a matter of weeks.
PTC’s battered shares shot up by more than 50% on the news.
NICE’s decision followed an accelerated approval of the drug in Europe pending the late-stage data. Regulators in Europe told FierceBiotech after the FDA’s rejection that it was planning to revisit that approval, but didn’t provide a timeline.
PTC has never produced statistically significant data to support the use of the drug for DMD cases spurred by nonsense mutations, about 15% of the total patient group. Their Phase II failed, and the Phase III flop came despite the fact that investigators designed it specifically to zero in on the positive aspects of the midstage study.
RBC’s Simos Simeonidis says that the approval by NICE must have come with a big discount on the price, given that the agency is known to play hardball with companies that have promising Phase III data. Also, he adds, investors may not want to bet that it stays on the market in Europe.
“Today’s news is definitely a positive for PTCT; however, what really matters for the stock, now that ataluren’s US future is essentially dead, is whether EMA will decide to keep the drug on the market or not,” writes Simeonidis. “We continue to believe that ataluren’s future in Europe is at serious risk and see at least a 50% probability that it is removed from the market, based on the failed Phase III trial.”
The FDA has already gunned down BioMarin’s ($BMRN) rival drug, and later this month an FDA panel will consider the case for Sarepta’s ($SRPT) eteplirsen. A harsh internal review of the drug inspired a recent rout in Sarepta’s share price, but with a delayed review and more data, there’s been some confidence that a strong show of support from families and friends might ultimately win the day.
If nothing else, PTC has proven again that regulators don’t always measure new drugs for Duchenne muscular dystrophy with the same yardstick used in other diseases.
- here's the release