Will the high-priced Hemgenix be reimbursed in Korea?

A one-shot hemophilia treatment for hemophilia B

The first and only one-time gene therapy for people with hemophilia B

CSL Behring is expected to apply for reimbursement next year… its high price of KRW 4.7 billion (based on U.S. price) is expected to act as a barrier

Company would need to appeal to the drug’s strengths such as its effect compared to lifelong treatment

The reimbursement listing process for Hemgenix (etranacogene dezaparvovec-drlb), a 'one-shot' treatment for hemophilia B that shows its effect as a single dose that received domestic approval recently, has been gaining attention in Korea.

 

Pic of Hemgenix

According to industry sources on the 21st, the Ministry of Food and Drug Safety (MFDS) recently approved CSL Behring Korea's hemophilia B gene therapy Hemgenix for the treatment of hemophilia B in adults.

 

Hemgenix is used to treat moderate-to-severe hemophilia B (congenital blood clotting factor IX deficiency) in adults without a history of Factor IX inhibitors.

 

Hemophilia is divided into two types, hemophilia A and hemophilia B, based on differences in the distribution of blood clotting factors VIII and IX, and the treatment approach differs depending on the type.

 

Hemophilia B is a congenital bleeding disorder caused by a single gene defect and is caused by a deficiency in coagulation factor IX (blood coagulation factor 9), a protein that helps blood coagulation and is mainly produced in the liver.

 

According to the 2019 White Paper on Hemophilia, the total number of hemophilia patients in Korea was 2,509, of which 1,746 (69.6%) were hemophilia A and 434 hemophilia B patients.

 

Hemgenix delivers a functional gene that activates the production of blood clotting factor IX, enabling patients to produce factor IX on their own, and can reduce bleeding continuously in the long term with just a single injection.

 

The recent availability of extended half-life therapies for hemophilia B has increased the convenience of prophylaxis.

 

While the standard half-life therapies required 104 days of administration per year (based on twice-weekly infusions), the newer extended half-life agents are administered once every 7 to 14 days.

 

However, the newer therapies have limitations despite the increased convenience due to the need for continuous treatment and the unavailability of a cure.

 

In this context, the company is expected to apply for Hemgenix’s reimbursement focusing on the fact that it only needs to be administered once.

 

According to industry sources, CSL Behring Korea is preparing to apply for reimbursement of Hemgenix.

 

The company is expected to apply as early as the first half of next year.

 

In the US, the drug is priced at KRW 4.7 billion (USD 3.5 million), making it virtually impossible for patients to pay on their own, so the company’s reimbursement challenge was naturally the next step.

 

A pharmaceutical industry insider said, “There is a consensus among industry insiders that it would not be easy for CSL Behring Korea to receive reimbursement due to Hemgenix’s very high price.

 

However, as the benefits provided by the drug are clear, the company will likely take a multifaceted approach for its reimbursement in Korea.” If and when the company applies for reimbursement, it is expected to emphasize Hemgenix’s role as a one-shot treatment.

 

While hemophilia B may not be considered life-threatening, patients can die from cerebral or organ bleeding, therefore the company may appeal to the drug’s value as a next-generation treatment that prevents the risks associated with regular intravenous infusions.

 

“Hemgenix offers a unique and innovative approach that enables patients to produce their factor XI, and can fundamentally change the hemophilia treatment paradigm,” said Ki-Woon Kim, CEO of CSL Behring Korea.

 

The issue is in its price.

 

CSL Behring Korea's other hemophilia B drug, Idelvion (albutrepenonacog alfa), is priced at KRW 1,800 per IU/vial, from the lowest 250 IU/vial to the highest 2,000 IU/vial.

 

Hemgenix’s dose is first determined based on age (below 12 or 12 years and older) and then applied a formula to determine the dosage based on body weight.

 

For example, an 80kg patient with severe hemophilia B whose levels are approximately 50% of the normal level would require 3,080 IU of treatment.

 

Simple calculation shows that this translates to 17.4 doses of Idelvion per year, which is a significant difference when comparing the U.S.

 

drug price, even when considering its lifelong use.

 

However, there are variables, such as the fact that the ultra-high priced drugs listed in Korea were given a price lower than those abroad.

 

“At this point, we believe the biggest hurdle in reimbursement will be the burden of drug price on Korea’s national health insurance finances,” said a pharmaceutical industry official.

 

“We expect CSL Behring Korea's track strategy and how convincing the therapeutic benefit and need of Hemgenix is to the government will be the key to its reimbursement in Korea.”