Novartis’ new gene replacement therapy is intended to help treat SMA Type 1. Credit: Novartis AG.

Novartis Pharma has received the Japanese Ministry of Health, Labour and Welfare (MHLW) approval for Zolgensma (onasemnogene abeparvovec) to treat spinal muscular atrophy (SMA).

The drug is for patients aged below two years, including individuals with a pre-symptomatic diagnosis.

SMA is a rare, genetic neuromuscular disorder that develops due to lack of a functional SMN1 gene. It causes irreversible loss of motor neurons and impacts motor functions.

Zolgensma is a one-time gene therapy that replaces the function of the missing or non-functional SMN1 gene. Given intravenously, the therapy delivers a working copy of the SMN gene into the patient’s cells.

Novartis Pharma president and representative director Kazunari Tsunaba said: “SMA is the leading genetic cause of infant death and, if left untreated in its most common form, Type 1, leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.

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“A one-time dose of Zolgensma has the potential to make a truly transformative impact on this life-threatening disease.”

The approval supports data from the Phase I START, START long-term follow-up, Phase III STR1VE-US, Phase III SPR1NT and Phase I / II STRONG clinical studies.

START and STR1VE-US assessed the safety and efficacy of Zolgensma in symptomatic SMA Type 1 patients aged less than six months at dosing with one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively.

Data revealed significant rates of survival, improvement in motor function, including the ability to sit without support. START long-term follow-up participants are now nearing five years of age.

Interim findings from the ongoing Phase III SPR1NT trial in pre-symptomatic patients showed a positive profile of the drug.

Last May, the US Food and Drug Administration (FDA) approved Zolgensma to treat children aged less than two years with SMA with bi-allelic mutations in the SMN1 gene.

Novartis subsidiary AveXis is also seeking approval for the drug in other markets, including Switzerland, Canada and Australia.