Pharmaceutical Market Europe • March 2026 • 7
NEWS
UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency
UCB’s Kygevvi (doxecitine and doxribtimine) has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to treat thymidine kinase 2 deficiency (TK2d) in adults and children, with symptom onset on or before 12 years of age.
The CHMP’s positive opinion is based on two studies of the treatment. The studies evaluated Kygevvi’s effect on survival, as well as on functional outcomes such as motor ability and the need for ventilatory and feeding support.
The most common side effects of Kygevvi were gastrointestinal disorders.
TK2d is an extremely rare genetic mitochondrial disease that causes severe and progressive muscle weakness.
The disease can affect activities such as walking, eating and breathing.
Patients who experience symptom onset on or before the age of 12 are at high risk for premature death, which often occurs within three years of symptoms appearing.
Estimates suggest that there are 1.64 cases of TK2d per million people globally. Aside from supportive care, there is currently no approved treatment for the disease in Europe.
US FDA approves Johnson & Johnson’s multiple myeloma treatment
The US FDA has approved Johnson & Johnson’s (J&J) Tecvayli (teclistamab) plus Darzalex Faspro (daratumumab and hyaluronidase) to treat adults with relapsed or refractory multiple myeloma (RRMM).
The approval is based on data from the ongoing, randomised, phase 3 MajesTEC-3 study.
The study is evaluating the safety and efficacy of teclistamab plus daratumumab versus standard of care in patients with RRMM who have received at least one previous treatment.
The treatment primes and activates the immune system to eradicate myeloma cells that express the BCMA protein.
Tecvayli plus Darzalex Faspro showed a statistically significant improvement of 83% in progression free survival and overall survival after three years in patients with RRMM compared to 30% with standard treatment.
This approval offers a potential new standard of care as early as second line and brings a novel treatment approach for the 40% of patients with multiple myeloma who experience disease relapse.
The results were presented in December 2025 as a late-breaking oral presentation at the American Society of Hematology Annual Meeting with simultaneous publication in The New England Journal of Medicine.
Lundbeck completes patient randomisation for phase 3 trial for multiple system atrophy treatment
The last patient in Lundbeck’s phase 3 clinical trial, MASCOT, has been randomised ahead of schedule in a study evaluating amlenetug as a potential treatment for multiple system atrophy (MSA).
The phase 3 trial will continue with a double-blind period where participants are randomised to receive either high or low doses of amlenetug, or placebo, for 72 weeks. This is followed by an optional open-label extension period where all participants will be offered treatment with amlenetug.
The multicentre phase 3 trial, which is currently ongoing across North America, Europe, Asia and Australia, aims to demonstrate that amlenetug has the potential to slow the clinical disease progression of MSA.
By addressing a key underlying cause of MSA by targeting the α-synuclein protein in the brain to inhibit its spread to nearby brain cells, amlenetug could become a first-in-class therapy for this rare disorder.
MSA causes debilitating damage to nerve cells in the brain. The symptoms are wide-ranging and include muscle control problems, with an average survival time after symptom onset of just over eight and a half years.