PTC Therapeutics (PTCT - Get Report) shares have doubled this morning on the surprising (I'd say shocking) news that European regulators recommended the conditional approval of Translarna as a new treatment for patients with Duchenne muscular dystrophy caused by so-called "nonsense" mutations.
Why is the PTC news a major surprise? Because the same European regulators rejected Translarna in 2013, telling the company the phase II study upon which the regulatory submission was based did not provide convincing proof of efficacy. PTC resubmitted essentially the same package of Translarna data again, and this time, regulators in Europe were satisfied enough to grant the drug conditional approval.
Here's what European regulators said today about their reversal:
Translarna was investigated in a pivotal study involving 174 patients with DMD. The results showed some evidence of efficacy of Translarna in slowing down the loss of walking ability in DMD patients. However, as part of the conditional marketing authorisation, the company will be required to provide comprehensive data on the efficacy of Translarna from an ongoing confirmatory study. Regarding safety, Translarna is generally well tolerated with the most common side effects being headache, nausea and vomiting.
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