Alexion Pharma Korea gained reimbursement from the Ministry of Health and Welfare for its atypical hemolytic uremic syndrome (aHUS) therapy Soliris (eculizumab) starting this month, the company said Monday.
A 30 mL bottle Soliris will cost around 6 million won ($5,371) starting Sunday.
Patients over 18 years of age should take 90 mL every week for four weeks, take 120 mL in the fifth week, and then take 120 mL every two weeks after that. Dosage will also depend on age and weight, according to the company.
Soliris first gained approval in January 2010 to treat paroxysmal nocturnal hemoglobinuria and gained an expanded indication to treat aHUS In March 2016.
aHUS is an extremely rare, life-threatening and progressive disorder caused by chronic and uncontrolled activation of the complement system, a branch of the immune system that destroys and removes foreign particles.
The condition is characterized by thrombotic microangiopathy (TMA) in which thrombosis and inflammations continue to damage small blood vessels throughout the body due to the chronic, uncontrolled activity of the complement system. It occurs regardless of age and gender and can cause major life-threatening complications such as acute renal failure, stroke, and heart failure.
Soliris is the first and only treatment to get indications for both rare diseases. The therapy is a terminal complement inhibitor that effectively blocks the uncontrolled activity of the complement system.
Handok retains exclusive rights to marketing and sales in Korea.
The reimbursement is based on four studies of aHUS patients. In four prospective, single-group studies of 100 patients taking Soliris for 26 weeks, the mean platelet level increased and remained within the normal range in all studies.
In 37 adults over 18 years of age with aHUS, who continually took Soliris for two years, more than 88 percent of patients maintained a normal lactate dehydrogenase (LDH) range, an indicator of normal platelet counts and cellular damage. The risk of co-mediated TMA was also reduced while early renal glomerular filtration rate improved significantly within seven days.
“As a pioneering biopharmaceutical company, Alexion is committed to providing dramatic therapies by providing innovative treatment options for patients with rare diseases left in a treatment blind spot,” said Alexion CEO Park Young-ae, noting that the reimbursement will dramatically change the lives of patients with the condition.