Russia registers multi-million-dollar gene therapy drug ‘Zolgensma’

The Russian Health Ministry has registered the world’s most expensive drug — a multi-million dollar gene therapy medication, sold under the brand name Zolgensma.

As first reported by the industry publication Pharmvestnik.ru on Thursday, December 9, the Russian Health Ministry issued a registration certificate for the drug that’s valid for five years.

Zolgensma, a one-time injection that costs upwards of $2 million, is used to treat spinal muscular atrophy (SMA). The drug’s manufacturer, the Swiss multinational pharmaceutical company Novartis, applied to register it for use in Russian in July 2020.

Also on Thursday, Novaya Gazeta reported that the Russian Health Ministry had put forward a proposal that would allow the Circle of Kindness Foundation to purchase registered medications before they go into general circulation.

• The Circle of Kindness Foundation was established by Russian President Vladimir Putin in early 2021, using revenue from an income tax increase for high-earning Russians. The foundation, which supports children with rare diseases, has purchased SMA drugs for around a thousand patients.
• Prior to Zolgensma’s registration, in August 2021, the Circle of Kindness Foundation announced that it had reached an agreement with Novartis on supplying the drug to Russia. The foundation underscored that the manufacturer had made “serious concessions” on the price. In September, the Circle of Kindness Foundation announced plans to purchase Zolgensma for 12 children.
• Zolgensma is the third SMA drug that the Russian Health Ministry has approved for use. Two other treatments — Spinraza, manufactured by the American company Biogen, and Evrysdi, manufactured by the Swiss company F. Hoffmann-La Roche — were registered in 2019 and 2020, respectively. Both Spinraza and Evrysdi cost hundreds of thousands of dollars and, unlike Zolgensma, require more than one dose.