One-time ￦2 billion therapy Zolgensma to be soon approved

MFDS completes safety and efficacy review of Zolgensma… whether reimbursement will be approved is key

Zolgensma (onasemnogene abeparvovec-xioi), a new gene therapy drug that attracted attention due to its high price of nearly 2 billion won per single shot, is soon to be approved in Korea.

 

As a spinal muscular atrophy gene therapy, Zolgensma is also called a ‘one-shot treatment’ as only a single dose is required during a patient’s lifetime.

 

As the drug is about to be approved, whether the drug will be allowed reimbursement is expected to be the key issue.

 

On the 26th, industry sources said that the Ministry of Food and Drug Safety (MFDS) completed the safety and efficacy review for Zolgensma.

 

The results are expected to be announced soon.

 

Zolgensma was designated as an orphan drug in December 2018 but was not formally approved in Korea.

 

The drug has gained much attention as a treatment for SMA that needs to be administered only once.

 

SMA is caused by a defect or mutation in the SMN1 gene that leads to a deficiency of motor neuron protein.

 

Without proper treatment, patients with SMA would need to depend on assistive devices and even result in death.

 

When administered, For Zolgensma, an SMN1 gene is inserted inside a virus vector, which is then injected into the patient's body to induce the production of SMN1 protein.

 

Various SMA treatments such as Spinraza (nusinersen, Biogen) and Evrysdi (risdiplam, Roche) have been recently released in Korea; however, the demand for the one-shot Zolgensma stays strong.

 

The issue at hand is its price.

 

The price of a single shot of Zolgensma is 2.5 billion won in the U.S.

 

and 1.89 billion won in Japan.

 

Experts say that the reimbursement standards in Korea are not sufficient to evaluate ultra high-priced drugs and that a new evaluation model needs to be designed for such drugs.

 

In a forum held on the 13th, Professor Hye-Young Kang of the Department of Pharmacy at Yonsei University said, “The optimal plan for each country should be prepared in consideration of each country’s history, culture, and economy.” She continued, “Adopting a reimbursement model from other countries may be dangerous as we cannot fully implement or understand the details or backgrounds of the models used in other countries.” On this, Yoon-Seok Yang, head of the Division of Pharmaceutical Benefits at MOHW said, “We can apply the current risk-sharing agreement scheme to evaluate the reimbursement of Zolgensma.” Explaining that the drug may be evaluated under current reimbursement standards, he added that “However, the payment structure needs to be deeply considered.” Meanwhile, Novartis’s ‘Kymriah,’ another ‘one-shot treatment’ indicated for the treatment of acute lymphoblastic leukemia (ALL), was approved in March and is in the process of being evaluated for reimbursement.