Ann: Trofinetide successful in Phase 2 trial in Fragile X, page-94

You wrote:

"Perhaps. EvaluatePharma analysis (2014) found that, contrary to accepted wisdom, there was no statistically significant difference in Phase 3 trial length between orphan and non-orphan drugs, with a median duration of 2.89 years for orphan drugs and 2.86 years for non-orphan drugs. Median FDA approval times were a bit shorter for orphan drugs -10 months for orphan vs. 13 months for non-orphans, due to orphans tending to receive Priority Review. The most significant difference was in cost – much less for orphans than for non-orphans."

Not sure on Phase 3 trial length or FDA approval times was referring to average time from Phase II to launch.

Specifically, Meekings KN, Williams CSM, and Arrowsmith JE analysis, the average time from Phase II to launch was 3.9 years for orphan drugs, compared with 5.4 years for non-orphan drugs.

http://csmres.co.uk/cs.public.upd/article-downloads/Meekings-Reuters-paper.pdf.

According to Bernstein Research, once a compound has been granted orphan designation, the odds for approval are high (82%) compared to traditional drugs (35%).

"Thanks for the example of Applied Genetics Technologies Corp. I’ll do some research and let you know what I find."

Thanks - look forward to hearing further on this.

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