CYTH - Cyclo gets positive EMA opinion on rare genetic disorder treatment development plan

Cyclo Therapeutics (CYTH) gets positive opinion from the Paediatric Committee ((PDCO)) of the European Medicines Agency ((EMA)) on its agreement with the proposed Paediatric Investigation Plan ((PIP)) for Trappsol Cyclo, currently in development for the treatment of Niemann-Pick Type C ((NPC1)), a rare genetic disease affecting 1 in 100K live births globally.The PIP opinion from PDCO has endorsed the clinical program to evaluate the safety, tolerability and efficacy of Trappsol Cyclo in patients from 3 years to less than 18 years of age with NPC Type C1.The opinion also backs the inclusion of a single-arm sub-study with patients from birth to less than 3 years of age with the condition.Cyclo Therapeutics would be eligible for up to an additional two years of marketing exclusivity in the EU, upon successful completion of the agreed PIP.

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Cyclo gets positive EMA opinion on rare genetic disorder treatment development plan