EGETIS THERAPEUTICS AB Egetis Therapeutics AB (publ) announced that the Company has submitted a protocol for an Expanded Access Program for Emcitate (tiratricol) in the USA, as requested by the Food and Drug Administration (FDA). Emcitate is under development for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency, a highly debilitating rare disease with no available treatment, but does not yet hold regulatory approval anywhere in the world. Until now, physicians treating MCT8-deficiency patients in the USA, who are not part of an ongoing clinical trial with Emcitate, have been able to request authorization to treat patients by submitting individual Investigational New Drug (IND) requests to the FDA.
As the number of diagnosed MCT8-deficiency patients has increased, with a concomitant increase in applications for individual INDs, Egetis has decided to implement an Expanded Access Program.
