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03 Feb 2026
Admin
News Article

EMA CHMP Backs UCB’s Kygevvi as First Potential Treatment for TK2 Deficiency

UCB announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization under exceptional circumstances for Kygevvi (doxecitine and doxribtimine).

The therapy is intended for paediatric and adult patients with genetically confirmed thymidine kinase 2 deficiency (TK2d) whose symptom onset occurred on or before 12 years of age.

The final European Commission (EC) decision is expected in Q2 2026.

A Milestone for an Ultra-Rare, Fatal Disease

TK2d is an ultra-rare, life-threatening mitochondrial disorder characterized by progressive and severe muscle weakness.

There are currently no approved disease-modifying treatments for TK2d in Europe beyond supportive care.

The disease:

Progressively impairs mobility, swallowing, and breathing
Often requires continuous caregiver support
Carries a high risk of premature death, especially in early-onset cases
Has an estimated global prevalence of 1.64 cases per million people

If approved, Kygevvi would become the first authorized therapy for TK2d in Europe.

Regulatory Pathway and Special Designations

Doxecitine and doxribtimine were supported under the EMA’s PRIME (PRIority MEdicines) scheme, which provides early regulatory support for medicines addressing high unmet needs.

Marketing authorization under exceptional circumstances is used when:

The disease is extremely rare
Comprehensive clinical data cannot reasonably be obtained
Ethical or practical barriers limit traditional trials

Clinical Evidence Supporting Kygevvi

Supportive evidence came from pooled data from two clinical studies in patients with genetically confirmed TK2d and symptom onset at or before 12 years.

The studies evaluated:

Motor milestone achievement
Need for ventilatory support
Need for feeding support
Overall survival

The most commonly reported side effects were gastrointestinal, including:

Diarrhoea
Vomiting
Abdominal pain

How Kygevvi Works

Doxecitine and doxribtimine are incorporated into mitochondrial DNA in skeletal muscle.

This mechanism:

Restores mitochondrial DNA copy number
Improves skeletal muscle function
Addresses the underlying metabolic defect in TK2d

What UCB Is Saying?

“This positive CHMP opinion marks a turning point in the treatment of TK2d,” said Donatello Crocetta, Chief Medical Officer at UCB.

“We are one step closer to bringing the first and only approved treatment for TK2d to the community.”

About UCB

UCB is a global biopharmaceutical company headquartered in Brussels, Belgium.

The company focuses on innovative medicines for immune system and central nervous system diseases.

Employees: 9,000+ across ~40 countries

2024 revenue: €6.1 billion

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