Following the US Food and Drug Administration refusing to file the letter for Translarna (ataluren), Jefferies’ analysts Gena Wang has upgraded the rating on the drug’s developer PTC Therapeutics from Underperform to Hold. He believes the chances of approval of the drug in Duchenne muscular dystrophy (DMD) in the US appear slim, although the EU decision is still unclear. He is lowering the PTC share price target from $18 to $12.
"With few precedents, we see reasonable probability of EMA withdrawing marketed ataluren based on prior mixed panel voting results," the analyst mentioned, while lowering the probability of success of the drug in DMD from 20 percent to 5 percent in the US and C.F. and from 50 percent to 30 percent in the EU/ROW,” said Mr Wang.
Also, Citi's Joel Beatty downgraded the rating on PTC from Buy to Neutral, while lowering the price target from $90 to $13. Mr Beatty believes that, due to this major setback, "the outlook for Translarna approval in the US looks poor," and that PTC Therapeutics would now need to "generate additional data before approval which could be expensive and risky."
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