Biogen attempts Qalsody reimb listing in Korea

New drug for a type of ALS without treatment options in Korea

Scheduled for submission to the insurance coverage subcommittee... key is in the government and pharmaceutical company reaching an agreement

Gains attention as a treatment option for an ultra-rare disease affecting just over 100 patients

The new ALS drug Qalsody (tofersen) has begun the process of applying for reimbursement coverage in Korea.

 

Since receiving domestic approval in August, the company has been moving rapidly to seek its reimbursement.

 

According to industry sources, Biogen Korea’s Qalsody, a treatment for amyotrophic lateral sclerosis (ALS) associated with SOD1 (Superoxide Dismutase 1) gene mutation, is scheduled to be reviewed by the Drug Reimbursement Standard Subcommittee.

 

Before approval, Qalsody was designated a GIFT (Global Innovative Products on Fast Track) item and as an orphan drug by the Ministry of Food and Drug Safety/ However, the road ahead is expected to be rocky due to its extremely high price.

 

In the U.S., Qalsody costs USD 15,097 per vial (approx.

 

KRW 22.03 million), and the annual treatment cost reaches USD 211,358 (approx.

 

KRW 308.5 million).

 

Biogen submitted a reimbursement application immediately after approval in August, but faced its first hurdle when reviewers requested additional data.

 

It now awaits the Reimbursement Standards Subcommittee review on the 28th.

 

Should the subcommittee require further review or additional data, the reimbursement schedule could face further delays due to re-review.

 

ALS, particularly SOD1 gene mutation ALS, falls into the category of ultra-rare diseases, with fewer than 100 patients worldwide.

 

Qalsody is the first targeted therapy developed specifically for this patient group.

 

Because ALS presents widely variable symptom onset patterns among patients, clinical trial design and endpoint determination are extremely challenging.

 

Experts note that the emergence of the first treatment option showing potential for improving disease progression, even in just one subset of patients, holds significant meaning in ALS, a field where drug development is exceptionally difficult.

 

Given the U.S.

 

Trump administration’s MFN drug-pricing stance and Korea’s historically restrictive approach to rare-disease reimbursement, experts warn that if reimbursement criteria become too narrow, the possibility of the drug's domestic launch being canceled cannot be ruled out.

 

It remains to be seen whether Qalsody will become a reimbursed treatment option in an area with virtually no therapeutic alternatives.

 

Meanwhile, in the Phase III VALOR study, Qalsody did not meet the primary endpoint, the ALSFRS-R functional score.

 

However, it demonstrated reductions in the secondary endpoints: a 26-38% decrease in total SOD1 protein cerebrospinal fluid and a 48-67% decrease in plasma neurofilament light (Nfl) concentration.