HAE treatment 'Takhzyro' closer to reimbursement

Has passed the Drug Reimbursement Evaluation Committee (DREC) of HIRA after receiving domestic approval in 2021

Demonstrated reduction in the number of HAE attacks through the Phase 3 trial…expected to become the new treatment option

Product photo of Takhzyro (lanadelumab)

A hereditary angioedema (HAE) treatment, 'Takhzyro,' appears closer to insurance reimbursement listing, approximately five years after acquiring domestic approval in South Korea.

 

Takeda Korea's Takhzyro (lanadelumab) has recently passed the Drug Reimbursement Evaluation Committee (DREC).

 

This is the first achievement since receiving approval from the Ministry of Food and Drug Safety (MFDS) in February 2021.

 

Attention has been drawn to whether Takhzyro will successfully undergo drug pricing negotiations with the National Health Insurance Service (NHIS) to become a new treatment option for the HAE area, where treatments are lacking.

 

HAE is a rare disorder caused by dysfunction of the C1-esterase inhibitor (C1-INH) protein, resulting in recurrent, unpredictable, and sudden swelling in different parts of the body.

 

Upper airway edema can restrict breathing and lead to asphyxiation.

 

Gastrointestinal tract swelling can cause emergency events, such as severe pain and intestinal obstruction.

 

Approximately 40% of patients have their first episode before age 5, and 75% before age 15.

 

However, most individuals undergo a 'diagnostic odyssey' and receive an accurate diagnosis only as adults.

 

This is why the number of diagnosed cases in South Korea was only 200-250 as of last year, despite an estimated total of approximately 1,000 patients.

 

In fact, it takes an average of 19 years to get diagnosed.

 

Even after the diagnosis, patients are exposed to unpredictable HAE attacks and emergencies.

 

However, in South Korea, patient access to treatments that provide fundamental care has been low.

 

Therefore, the medical field has been consistently requesting reimbursement listing for Takhzyro.

 

It remains to be seen whether drug price negotiations with the NHIS for Takhzyro will be successfully concluded and emerge as a new treatment option for HAE, where therapeutic agents are scarce.

 

Meanwhile, Takhzyro demonstrated efficacy in the Phase 3 HELP study.

 

The study results showed that the group receiving Takhzyro every two weeks reduced the average monthly attack frequency of HAE attacks by 87% compared with the placebo group, while the group receiving Takhzyro every four weeks achieved a 74% reduction.