The right age to use KRW 2.5 billion SMA drug Zolgensma

Novartis says children under 2, Central Committee says “younger”

Novartis

Prior to the South Korean health authority’s approval on Zolgensma, known as the most expensive drug in the world, Central Pharmaceutical Affairs Deliberation Committee discussed the appropriate age of the drug user.

 

The manufacturer Novartis has applied for an indication to treat patients under the age of two, but the committee concluded the age bracket should be lowered due to lack of clinical data.

 

Unlike the U.S.

 

and Japanese health authorities indicated the drug to be used on children under age two, the Korean health authority would likely to indicate the drug to treat infants under one year or six months.

 

The Ministry of Food and Drug Safety (MFDS) disclosed the minutes from Central Pharmaceutical Affairs Deliberation Committee meeting in last May discussing the designation of Zolgensma as an orphan drug.

 

Developed by Novartis, Zolgensma is a spinal muscular atrophy (SMA) treatment and the company priced the drug initially at 2.5 billion won, which gave the drug the title of ‘the most expensive drug in the world.’ The U.S.

 

Food and Drug Administration (FDA) granted approval on the drug in May last year.

 

In Korea, Novartis Korea has submitted the health authority approval application in last February and it awaits the final approval.

 

SMA is an autosomal recessive genetic disorder caused by mutations in survival motor neuron 1 (SMN1) gene that encodes the SMN protein.

 

It is a severe disease that usually occurs in infants, which leads to death.

 

The diagnosed patients are in dire need of treatment at the moment.

 

Categorized into three types, the two-thirds of type 1 result in death before the age of two, and type 2 patients may survive until pre-school, but most of them needs assistance in wheelchair due to muscular disability.

 

Infants with type 3 seem normal but the disease advances slowly.

 

At the moment, the existing SMA treatment in the market is Biogen’s Spinraza.

 

Approved in 2017, Spinraza was also a vastly expensive drug priced at about 100 million won per administration.

 

Although Spinraza has to be administered three to four times a year for a number of years, Zolgensma is a gene therapy administered only once.

 

In last May, Central Pharmaceutical Affairs Deliberation Committee reviewed the subject patient’s age and treatment initiation period as requested by Norvatis.

 

The company suggested using the drug on SMA patient under the age of two, but the committee members were against it.

 

A committee member noted, “For clinical purposes, a variety of options is always welcomed, but Spinraza has been developed and the government should carefully decide whether to indicate the drug for ‘age under two’ or not, as the long-term clinical data on the drug’s efficacy and safety are insufficient.” Another committee member explained, “It would seem more appropriate to set the subject patient’s age as under one year or six months.” The chair of the committee also said the manufacturer’s request was not appropriate, so the patient age and type should be limited more.

 

Instead of conventionally deciding the treatment period based on the types, the committee saw it would be appropriate to use a new categorization to administer the drug in patient with or without the symptom (diagnosed by gene testing).

 

The committee also urged MFDS to consider the drug significantly improving the convenience of administration method as a gene therapy, although it cannot clearly prove improvement in safety and efficacy compared to the existing treatment option.

 

Currently, about 17 patients under the age of two are diagnosed with SMA annually in Korea.

 

The next concern for the drug is its high price.

 

As the reimbursement listing process for Spinraza was a long and tough race, the health insurance authority would also have to expect the world’s most expensive Spinraza to bring even a tougher process.