Stu Peltz from PTC Therapeutics hosted a call today regarding the Refuse to File letter from the FDA, dated February 22nd. More detailed information is available in the transcript from the earnings call on February 29.
Stu explained the two concerns raised by the FDA:
1) There was not evidence that this drug would not create an addiction since it crosses the blood-brain barrier. PTC does not feel this is an issue and can be answered.
2) The Intent to Treat (total trial group) patients did not exhibit statistically significant benefit over the placebo group. PTC felt that their pre-specified sub-group analysis which showed significant benefit would have been accepted by the FDA, but the FDA considered it a post-hoc analysis.
They believe they should have an AdCom meeting where the community can express their opinion. They will reach out to community after clarification from the FDA for those who are anxious to support them. PTC expects EU will continue with the approval since they got conditional approval after the previous failed trial. The kids currently on drug will continue on drug and the younger patient trial should continue as planned.
Some parents of boys currently taking the drug expressed the benefit they saw from ataluren. PTC will check into the data generated from the trial subjects who participated in the imaging study in Florida.