On November 3rd UCB announced that the US Food and Drug Administration (FDA) had approved Kygevvi for the treatment of Thymidine kinase deficiency (TK2d) in adults and pediatric patients 12 years of age or older. It is the world’s first and only approved treatment for this patient population.
TK2D is an extremely rare and life-threatening inherited mitochondrial disease characterized by progressive (progressive) severe myasthenia gravis (myopathy) that has not been previously treated with approved drugs except for symptomatic supportive care. The disease often leads to an early death, and the risk of death is high for patients who develop their first symptoms at age 12 or earlier (early death usually occurs within about 3 years of symptom onset) . The estimated global prevalence of TK2D was 1.64 cases per million people (range, 0.5-3.1) .
“The approval of Deoxycytidine and deoxythymidine is a critical moment for the TK2D patient population, and prior to that there were no FDA-approved treatments for TK2d other than supportive [ palliative ] treatment,” said Donatello Crocetta, head of medical affairs and Chief Medical Officer at the company. “We are grateful to all the patients, families and friends, advocates, health care workers, and the team and staff who worked so hard for this clinical trial to share this important journey with us.”
“The importance of this FDA approval can not be overstated for patients and their families diagnosed with TK2D. This population of super-rare diseases urgently needs available treatment options. Carers and families have had to bear a heavy burden of Disease for a long time,” said Kristen Clifford, president and chief executive of the United Mitochondrial Disease Foundation. “The first FDA-approved TK2D treatment in this patient population not only meets critical medical needs but also offers hope for life.”
“I have been working on mitochondrial disease for more than 30 years and have seen first-hand the dramatic impact TK2D has on patients and families. The long-awaited approval of treatment is a significant step forward in supporting and managing this serious disease,” said Dr Michio Hirano, professor of neurology and director of Myopathology at Columbia University Irving Medical Center.
“The company is exploring policy mechanisms such as ‘trial first’ and emergency clinical use to speed up the introduction of innovative drugs to meet specific clinical needs and shorten waiting times for patients,” said Ma Yajun, general manager of the company in China. “We hope that with continued efforts, Chinese patients will be able to benefit earlier from the latest advances in global medical technology.”