Zolgensma Proposed to Cost 167 Million Yen in Japan

TOKYO, Japan – Japanese media have reported that the price of Novartis’ spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec) is expected to be 167 million yen under the National Health Insurance (NHI) scheme.

Pending approval of its price by the Central Social Insurance Medical Council, or Chuikyo, Zolgensma would become the first drug with a price to exceed 100 million yen covered by the NHI.

In Japan, Zolgensma is approved for the treatment of SMA in patients aged 2 years and under who are pre-symptomatic at diagnosis and test negative for elevated anti-AAV9 antibodies.

The gene therapy was approved in March of this year by the Ministry of Health, Labor, and Welfare (MHLW) following sakigake fast-track designation in March 2018.

Japan currently has no pricing rules for cell and gene therapies, which thus far have been categorized as “regenerative medicine products,” and instead used existing drug or medical device rules, depending on what each given innovative therapy does. For example, Novartis’ CAR T-cell therapy Kymriah (tisagenlecleucel) was priced via Japan’s cost-based method and initially, upon approval, was granted a 35% utility premium for its anti-tumor effect and 10% marketability premium for its orphan drug designation.

In the case of Zolgensma, health official insiders from the MHLW suggested that the price of Zolgensma was established by the comparator method, its cost compared to that of Biogen’s Spinraza (nusinersen), another groundbreaking treatment for SMA. Additionally, Zolgensma would receive the utility premium, which awards its high efficacy, safety, and improvement over existing treatments, and a sakigake premium.

It is expected that Zolgensma will be administered to around 25 patients annually once it is covered by the NHI.

The price for Zolgensma is not yet confirmed. Once it is, and analysts have verified the amount in our source, the price will be reflected in Pricentric One.