Same ‘one-shot’ but different? Zolgensma reimb slow

Not deliberated by Drug Reimbursement Evaluation Committee (DREC) in February… agenda is left pending for a year now

Excellent in its efficacy… demonstrates survival benefit without ventilators

Although both drugs are from the same pharmaceutical company and are both one-shot, high-priced drugs, the two drugs are showing a stark difference in their results.

 

Discussions on the reimbursement listing for Novartis Korea’s Zolgensma (onasemnogene abeparvovec) is not progressing so smoothly.

 

The company had applied for the reimbursement listing of its Spinal Muscular Atrophy (SMA) treatment Zolgensma in May last year through the approval-benefit appraisal linkage system, but the agenda has not been deliberated by the National Health Insurance Service DREC until now.

 

As it is a rare disease drug, the Drug Reimbursement Standard Subcommittee must set reimbursement standards, and the drug pass DREC deliberations to receive reimbursement.

 

According to industry sources, the government had numerously requested data supplementation to the company, upon which the company repeatedly submitted additional data.

 

Originally, drugs should receive DREC deliberations within 150 days of submitting an application under the National Health Insurance Act, but this period has long been exceeded.

 

This is different from the results made for the company’s new CAR-T therapy ‘Kymriah (tisagenlecleucel).’ Kymriah has passed DREC deliberations last January.

 

Last month, Yong-Myung Jang, HIRA’s Director of Development at the Health Insurance Review and Assessment Service (HIRA) mentioned Zolgensma at a press meeting with the Special Press Corp last month, raising hopes on the progress to be made for Zolgensma.

 

Director Jang said, “We have collected opinions from the society, experts, and held an expert advisory meeting to discuss its clinical use.

 

DREC deliberations are in the way for the drug.” However, Zolgensma was not on the list for deliberations in February.

 

Meanwhile, Zolgensma is a gene therapy that contains a genetic material that functionally substitutes defective genes.

 

The Ministry of Food and Drug Safety approved Zolgensma as the second advanced biologic product after Kymriah.

 

Advanced biopharmaceuticals are cell therapies or gene therapies that use live cells, tissues, or genetic material as ingredients.

 

Under the ‘Safety and Support Act for Advanced Regenerative Medicine and Advanced Biopharmaceuticals,’ advanced biopharmaceuticals can receive differentiated safety management including long-term follow-up studies and support for R&D and product commercialization.

 

Despite being a one-shot treatment, the price of the single shot costs 2.5 billion won in the U.S.

 

and 1.89 billion won in Japan.

 

Due to the high price, the listing process for Zolgensma in Korea is also expected to walk a rocky road However, expectations for its efficacy are very high.

 

The Phase III SPR1NT and STR1VE-EU results for Zolgensma that was presented recently gained much attention.

 

In the SPR1NT study, all pediatric SMA patients with two SMN Type 2 gene copies (Cohort 1) that were treated presymptomatically survived without requiring ventilatory or nutritional assistance and achieved sitting independently for 30 seconds or more.

 

Most (11/14) patients achieved age-appropriate motor milestones within the World Health Organization (WHO) window of normal development.

 

In the STR1VE-EU study, most pediatric patients (82%) that were treated with Zolgensma, including those with severe SMA, achieved motor milestones unseen in the natural history of SMA Type 1.

 

An official from the company said, “Patients and their families are longing for the prompt reimbursement of Zolgensma.

 

We are working closely with relevant ministries so that we can receive reimbursement as soon as possible and not deprive the opportunity for patients desperately waiting to be treated with Zolgensma.”