Thursday, April 12, 2018 11:14:59 AM
See below from Neuren website, Product Development page (bold is mine). Neuren is way ahead of AVXL and rapidly advancing to Phase 3.
Neuren conducted a Phase 2 double-blind placebo-controlled clinical trial in girls aged 5 to 15 years with Rett syndrome, in which statistically significant and clinically meaningful improvement was demonstrated. The benefit was observed in both clinician and caregiver assessments. This followed positive trends observed in Neuren's earlier Phase 2 trial in adolescents and adults aged 16 to 45 years.
The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted Orphan Drug designation to Neuren for trofinetide in Rett syndrome. The FDA has also granted Fast Track designation.
Rettsyndrome.org (International Rett Syndrome Foundation, or IRSF) has provided advice to Neuren on clinical trial strategy, introductions to leading clinical investigators and grant towards the cost of Neuren’s Rett syndrome trials. Support from an advocacy organisation such as IRSF in discussions with the FDA and communications with patients, families and investigators is an important factor assisting with the successful implementation of Neuren’s development programs.
Neuren conducted a Phase 2 double-blind placebo-controlled clinical trial in girls aged 5 to 15 years with Rett syndrome, in which statistically significant and clinically meaningful improvement was demonstrated. The benefit was observed in both clinician and caregiver assessments. This followed positive trends observed in Neuren's earlier Phase 2 trial in adolescents and adults aged 16 to 45 years.
The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted Orphan Drug designation to Neuren for trofinetide in Rett syndrome. The FDA has also granted Fast Track designation.
Rettsyndrome.org (International Rett Syndrome Foundation, or IRSF) has provided advice to Neuren on clinical trial strategy, introductions to leading clinical investigators and grant towards the cost of Neuren’s Rett syndrome trials. Support from an advocacy organisation such as IRSF in discussions with the FDA and communications with patients, families and investigators is an important factor assisting with the successful implementation of Neuren’s development programs.
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