Expansion of coverage for SMA treatment Spinraza

Differentiation from competing drugs

Biogen Korea holds a press conference to commemorate Spinraza's expansion on the 2nd

Spinraza, age restriction under 3 years removed from dosing standards for spinal muscular atrophy patients

Biogen Korea held a press conference on the 2nd at the Conrad Hotel in Yeouido, Seoul to commemorate the expansion of the reimbursement standard for Spinraza, the first SMA treatment developed

Differentiation between single-dose Zolgensma and oral Evrysdi “Spinraza welcomes relaxation of salary standards” Hyeongjun Park, Professor of Neurology at Gangnam Severance Hospital, gave a presentation on changes in the SMA treatment environment.

 

Biogen's Spinraza, a treatment for spinal muscular atrophy (SMA) with the advantage of direct intrathecal administration and multiple administration, succeeded in easing the age limit for reimbursement.

 

It showed the potential to differentiate itself from competing drugs such as Novartis' Zolgensma, a one-shot treatment, and Roche's Evrysdi, a PO drug.

 

Biogen Korea held a press conference on the 2nd at the Conrad Hotel in Yeouido, Seoul to commemorate the expansion of the reimbursement standard for Spinraza, the first SMA treatment developed.

 

In order to receive Spinraza coverage in Korea, patients with 5q SMA must be 5q ▲ genetically diagnosed with a deletion or mutation in the SMN-1 gene ▲ not using a permanent ventilator ▲ develop SMA-related clinical symptoms and signs under the age of 3 All criteria had to be met.

 

Due to this benefit expansion, the age limit for children under 3 years old has been deleted.

 

As a result, SMA type 3 patients who developed symptoms after the age of 3 can now begin treatment with Spinraza for which reimbursement is applied.

 

This expansion of the reimbursement standard is considered to be one step ahead of competing drugs in dominating the SMA treatment market.

 

Zolgensma, a one-shot treatment, works by inserting a functional replacement copy of the SMN1 gene into a carrier called a ‘vector’ and then delivering it to motor neurons in the body through intravenous injection.

 

The disadvantage is that it is difficult to administer multiple doses to patients who cannot be treated with a single administration.

 

Evrysdi, an oral medication, has a similar mechanism to Spinraza but has the disadvantage of not being able to be administered directly intrathecally.

 

Park Hyeong-jun, a professor of neurology at Gangnam Severance Hospital, said, “Zolgensma can only be administered once, so if the patient has experienced the virus once, an immune response will occur, making multiple administrations difficult.” He added, “Due to the risk of an immune response, it is only administered to patients under 2 years of age.” He continued, “In the case of Evrysdi and Spinraza, there is no direct comparative study, so it is difficult to say which drug is better.

 

The difference is that Spinraza can be administered directly intrathecally.

 

Because Evrysdi is an oral medication, there is no guarantee that the drug will only go to the spinal cord when administered.

 

“There may be a shortage of doses,” he added.

 

He said that it is meaningful to see improvement in symptoms in a wider range of patients by lifting Spinraza's age restriction.

 

Professor Park said, “It was getting worse, but it’s important to feel like it’s improving.

 

The probability of a patient who cannot walk walking and a person lying down and rolling to the side are different.

 

There are many patients whose movements gradually improve.

 

“This is why we need Spinraza, a multi-dose drug,” he said, adding, “It is true that people are cautious because Spinraza is a drug that costs more than 10 million won per bottle, but it is meaningful that the scope of what can be done for patients has expanded due to this expansion of coverage.” Ultra-rare disease SMA, the advantage of Spinraza is that can be administered directly.

 

SMA is a rare, genetic, neuromuscular disorder whose symptoms are caused by a deficiency of the survival motor neuron (SMN) protein caused by a damaged or missing SMN1 gene.

 

People with SMA may have difficulty sitting, standing, and walking.

 

SMA is a leading cause of death in infants and young children, affecting approximately 1 in 10,000 live births, and causes a variety of disabilities in patients older than their teens.

 

It is classified into types 1 to 4 depending on the time of symptom onset.

 

Spinraza is an antisense oligonucleotide (ASO) with a mechanism of action that continuously increases the amount of SMN protein.

 

In order to deliver the treatment to the cause of the disease, it can be administered directly to the central nervous system where motor neurons are located through intrathecal injection therapy.

 

Spinraza can be differentiated in administration method because it can be administered multiple times.

 

The treatment has confirmed its continued effectiveness and safety profile across all ages and types based on clinical research data and real-world use evidence (RWE) accumulated through treatment for up to 8 years or more.