3 more successful Zolgensma treatment cases reported…

19 of 20 cases show improvement

Post-reimbursement evaluation results of the KRW 2 billion ‘one-shot’ ultra-high-priced drug

4 reports disclosed... only 1 case of failure was reported

Zolgensma, a one-shot treatment for spinal muscular atrophy (SMA), is demonstrating a high effect post-reimbursement.

 

The government has been conducting a post-marketing performance evaluation on the drug since its reimbursement in August 2022, and so far, there has been only 1 case of failure reported after its use.

 

The drug costs about KRW 2 billion per dose off-label, but the patient's out-of-pocket cost for the single dose has been lowered to a maximum of KRW 5.98 million (applied the 10% co-payment rate) with insurance reimbursement.

 

According to the performance evaluation results on Zolgensma Inj that were released by the Health Insurance Review and Assessment Service on Sept.

 

29, all the 3 patient cases reported showed significant improvement.

 

A 20-month-old boy, a 2-month-old girl, and a 2-month-old boy who received treatment with Zolgensma all showed an increase in motor function test scores.

 

As a result, HIRA concluded that a significant improvement occurred after the drug’s administration.

 

Patients who receive Zolgensma are required to be clinically evaluated before administration and every 6 months thereafter for up to 5 years.

 

Objective data such as medical records on the patient need to be submitted to HIRA for clinical evaluations.

 

Failure is defined as a) use of permanent ventilation or death; b) when the CHOP-INTEND score does not improve by at least 4 points from the pre-dose baseline; or c) even if the improvement stated in b) is achieved, the CHOP-INTEND score decreases by at least 4 points or HFMSE decreases by at least 3 points in 2 consecutive response evaluations.

 

However, there has only been 1 case of failure among the performance evaluations disclosed by HIRA so far.

 

The single treatment failure case of a 2-year-old girl was released in the first evaluation data that was disclosed in June of last year.

 

At the time, the patient had respiratory problems due to SMA and died of acute respiratory failure.

 

Other than the single case, patients in all of the other presented outcomes have shown significant improvement.

 

19 of the 20 cases in the 4 published reports to date have shown meaningful improvement.

 

Zolgensma is evaluated to be superior to its alternative – the intrathecal injection alternative Spinraza (nusinersen) - in terms of administration method, and superior in event-free survival and motor function achievement when indirectly compared to its alternative in patients with rapidly progressive SMY Type 1 patients.

 

Despite its very high price of KRW 2 billion, the drug is proving to be effective, justifying its reimbursement coverage.