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A RETROspective Study of the Combination of Pyrimidine Nucleos(t)Ides in Patients With Thymidine Kinase 2 Deficiency (TK2)

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Trial Profile

A RETROspective Study of the Combination of Pyrimidine Nucleos(t)Ides in Patients With Thymidine Kinase 2 Deficiency (TK2)

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 12 Feb 2026

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At a glance

  • Drugs Deoxycytidine/deoxythymidine (Primary)
  • Indications Mitochondrial disorders
  • Focus Pharmacogenomic; Therapeutic Use
  • Acronyms RETRO
  • Sponsors Modis Therapeutics; Zogenix

Most Recent Events

  • 30 Jan 2026 According to an UCB media release, based on pooled data from two studies, NCT03845712 and NCT03701568, the CHMP of the EMA adopted a positive opinion recommending granting marketing authorization under exceptional circumstances for KYGEVVI (doxecitine and doxribtimine) for the treatment of pediatric and adult patients with genetically confirmed thymidine kinase 2 deficiency with an age of symptom onset on or before 12 years. The EC decision is expected in Q2 2026.
  • 03 Nov 2025 According to an UCB media release, company announced that a regulatory review of doxecitine and doxribtimine is currently underway by the EMA (European Medicines Agency), and further regulatory submissions are planned. KYGEVVI is currently not approved for use in any indication by any regulatory authority outside of the U.S. UCB expects KYGEVVI to be commercially available in the U.S. in Q1, 2026.
  • 03 Nov 2025 According to an UCB media release, company announced that KYGEVVI has been granted approval by the U.S. Food and Drug Administration (FDA) for the treatment of adults and pediatric patients living with thymidine kinase 2 deficiency (TK2d).

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