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Egetis is an innovative, unique, and integrated pharmaceutical drug development company, focusing on projects in late-stage development for treatment of serious rare/niche diseases with significant unmet medical needs in the orphan drug segment

Egetis receives approval in Turkey for compassionate use of Emcitate® for MCT8 deficiency

September 9, 2021

Egetis Therapeutics AB (publ) (ticker: EGTX) today announced that it has received approval from the Turkish Medicines Agency for a compassionate use program of its leading candidate drug Emcitate to treat the rare genetic disease MCT8 deficiency.

Compassionate use and named patient programs are mechanisms to allow early access to a medicine prior to regulatory marketing approval, granted to pharmaceuticals under development for situations with high unmet medical needs and where no available treatment alternatives exist or are suitable.
 
Compassionate use programs, such as the current Turkish example, need to go through a detailed data review process by the national medicines agencies to become approved. Compassionate use programs are initiated by pharmaceutical companies and allow access to a group of patients in a certain country, when the treating physician considers it appropriate.
 
Outside Turkey, Emcitate is already being supplied on a named patient basis, following individual regulatory approval from the national regulatory agencies, to more than 120 patients in over 20 countries.
 
“I am happy that we now have a solution in place to be able to supply this promising therapy to the MCT8 patients also in Turkey under a compassionate use program. The approval from the Turkish Medicines Agency is another important step towards our goal to secure access to Emcitate to MCT8 patients across the globe and also verifies the substantial unmet medical need and interest to treat patients that suffer from MCT8 deficiency with Emcitate” said Nicklas Westerholm, CEO, Egetis Therapeutics.

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