Zolgensma, a treatment for spinal muscular atrophy (SMA), which is said to be the most expensive drug in existence, is finally landing in Korea.
Novartis’ rare disease gene therapy Zolgensma (Ingredient: Onasemnogeneabeparvovec) is an ultra-high priced drug that costs KRW 2.5 billion (about $2.12 million) in the U.S. and KRW 1.89 billion in Japan per dose.
Zolgensma was designated as an orphan drug in Korea in December 2018, and an approval application was submitted to the Ministry of Food and Drug Safety in December 2019, but no news was heard until about a year and a half.
There is also a treatment for spinal muscular atrophy in Korea: Biogen’s Spinraza (Ingredient: Nusinersen Sodium) and Roche’s Evrysdi (Ingredient: Risdiplam).
In the case of Spinraza, health insurance can be applied through a ‘pre-approval system’ that determines where to receive benefits by cases.
Zolgensma is known as a 'one-shot cure' that treats SMA with just one dose, so there were patients waiting for its release.
Meanwhile, the Ministry of Food and Drug Safety has approved Zolensma on 28th May.
According to the FDA’s permission, Zolensma may be used in patients with a double allelic mutation in the Survival Motor Neuron (SMN 1) gene ▲three or fewer copies of the SMN Type 2 gene ▲clinical diagnosis of type 1.
The dosage varies depending on the patient’s weight, and re-administering is not allowed.
With its approval in Korea, the possibility of insurance benefits is now focusing.
However, due to its high-priced tag, the authorities are extremely cautious about applying health insurance to Zolgensma.
