Zolgensma was added to target diseases

Contrary to the negative opinion of the Central Pharmaceutical Affairs Review Committee, most of Novartis' changes were accepted

The difference in benefit size is large

The minutes of the Central Pharmaceutical Affairs Review Committee were released in connection with the expansion of the target age of the expensive drug 'Zolgensma' of ₩2.5 billion, and it was revealed that the target disease was added on the 3rd.

 

The committee presented a negative opinion on the expansion of the age group, but Orphan drug designation change announcement revealed that the age group has expanded.

 

At the beginning of this year, Novartis Korea, a sales company of this drug, applied for a formal approval, and it is noteworthy how the indications will be determined when the product is approved.

 

This is because, as this drug is an ultra-high-priced drug with a price of ₩2.5 billion by the manufacturer, the difference in the amount of benefits in the future increases depending on the difference in the number of patients.

 

According to the industry on the 13th, the MFDS has further expanded the target disease of Zolgensma, currently designated as an orphan drug on the 3rd.

 

Zolgensma was designated as an orphan drug in December 2018 in Korea.

 

When designated as an orphan drug, approval screening is accelerated, and purchases are facilitated through the KOEDC.

 

It was designated as the first orphan drug, the target disease was announced as 'spinal muscular dystrophy (Type 1)'.

 

However, the target diseases announced this time have been changed to ▲if there is a clinical diagnosis of type 1, and ▲the number of copies of the Survival Motor Neuron 2 (SMN2) gene is 3 or less Survival Motor Neuron 1 (SMN1)' in patients with Spinal Muscular Atrophy (SMA) with a double allelic mutation in the gene.

 

Spinal muscular dystrophy is all about patients with mutations in the SMN1 gene that act on the motor nerves.

 

However, it is divided into 3 types according to symptoms.

 

Type 1, which was previously a target disease, dies within 2 years of age from 2/3 or more, and type 2 can survive until elementary school entrance, but there are many cases of using a wheelchair due to muscle disorder, and type 3 appears normal in the neonatal period, but progresses slowly.

 

In particular, type 1 is mostly diagnosed in infants under 6 months.

 

Zolgensma can be administered to children under the age of 2 in the United States and Japan, and to children under 21 kg of body weight, regardless of age, approved earlier this year in Europe.

 

On the other hand, since the use of TYPE 1 for orphan drugs designated in Korea is limited to infants under 6 months, Novartis tried to expand the patient group by adding diseases for orphan drugs before official approval.

 

However, at the committee held in May, it was desirable to further restrict the target age, type, etc., and presented a negative opinion on the contents of Novartis' application.

 

However, the 'survival motor neuron 2 (SMN2) gene's copy number of 3 or less' is also included in the target disease, resulting in an expanded age group.

 

This is because many patients with type 2 and type 3 SMA diagnosed after 6 months have 3 to 4 SMN2 genes.

 

Patients with type 1 have only one or two SMN2 genes.

 

Currently, 60% of SMA patients are in type 1, 30% in type 2, and 10% in type 3.

 

It means that Zolgensma can be used in types 2 and 3 regardless of age.

 

However, it is difficult to predict the final indication as this drug has not yet been officially approved in Korea.

 

However, there is a high possibility that the designation of orphan drugs will be reflected in the official approval.

 

From a manufacturer's point of view, sales can be expected to increase due to the expansion of the age group.

 

On the contrary, it seems that the insurance authorities' concerns will increase in the phase of applying for benefits after formal approval.

 

This is because even if the number of patients used increases by just one or two, the size of the health insurance premium differs by several billion won(₩).

 

Currently, about 17 SMA patients under the age of 2 in Korea are known to have been diagnosed annually.

 

When the manufacturer's suggested amount is applied to the domestic patient group, the size of the treatment will reach ₩42.5 billion per year.