Zolgensma shows 94% effect during post-evaluations

… only 1 out of 18 patients fail treatment

17 showed significant improvement...mostly in infants and young children

The spinal muscular atrophy (SMA) treatment Zolgensma (onasemnogene abeparvovec) showed promising results suited to its high price.

 

The use of the once-daily, one-shot treatment, which has an insurance cap set at KRW 2, requires a post-marketing evaluation to measure its cost-effectiveness.

 

According to industry sources on the 5th, Zolgensma Inj was approved for reimbursement in August 2022 and was administered to 21 patients by November last year, 18 of whom have received outcome evaluations.

 

17 of the 18 patients showed significant improvement, with 1 patient failing to receive the drug due to death.

 

The Health Reviews and Assessment Service recently released the results of the Zolgensma Inj performance evaluation that contained the results above.

 

The performance evaluations for Zolgensma Inj are conducted before dosing and every 6 months following administration for up to five years.

 

Failure was defined as: a) permanent ventilation or death; b) CHOP-INTEND score not improving by at least 4 points from pre-dose baseline; or c) a decrease in CHOP-INTEND of at least 4 points or HFMSE of at least 3 points on two consecutive response assessments, even if the b) improvement was achieved.

 

However, a significant improvement after treatment was considered to have occurred if there was an increase in motor function test scores, even if the patient’s outcome falls within the criteria for treatment failure.

 

Spinal muscular atrophy is a genetic disorder caused by problems in the part of the nervous system that controls the movement of the voluntary muscles.

 

Zolgensma is designed to deliver a functional copy of the SMN1 (survival motor neuron 1) gene into cells via transfection.

 

It promotes the survival and function of the transgenic motor neurons by alternatively expressing the SMN protein in the motor neurons.

 

The majority of patients receiving Zolgensma are infants less than 24 months old.

 

The 9 additional outcomes reported by the panel were also all infants at 24 months of age.

 

Eight of these patients showed significant improvement from 12 to 24 months.

 

One patient showed a decrease in HFMSE of more than 3 points at the 24-month evaluation and was required to confirm motor function via HFMSE at the next review, and if necessary, submit video evidence to confirm the HFMSE score.

 

A total of 21 patients had received Zolgensma Inj by November of last year when the post-evaluation was conducted, the agency said.

 

Of these, the results of 18 patients were disclosed because 3 are yet to receive the 6-month post-evaluations.

 

One of the 18 patients showed treatment failure with death, and 17 showed significant improvement, rendering the effectiveness rate to 94.4%.

 

As high as the drug’s price is, with an insurance price ceiling set at KRW19.817,269, the drug has been showing a clear effect.

 

The patient’s out-of-pocket cost for the drug is KRW 5.98 million (10% coinsurance rate) for a single dose with health insurance.