In its decision, the Russian Health Ministry noted that much more data exists on Spinraza’s benefits for patients 17 years and younger than for those 18 and older. But it did not restrict the use of Spinraza based on age or disease severity, according to Olga Germanenko, president of the SMA Family Foundation Russia, which pushed for approval.
The next step in bringing Spinraza to the estimated 2,000 to 3,000 Russians with SMA will be to persuade government officials and lawmakers that Russia’s national health insurance system should cover it, Germanenko told SMA News Today in a phone interview. The foundation also plans a campaign to try to rev up doctors’ enthusiasm for Spinraza, she said.
“Approval was a great step forward, but there is a lot to be done before the government decides to reimburse for Spinraza,” she said, adding that the journey to full insurance coverage of the costly drug will be long. The U.S. retail cost of Spinraza is $750,000 the first year, and $375,000 each year thereafter.
Spinraza authorization came Aug. 16, a month after Russian doctors began administering the therapy to 40 children with SMA type 1 — a severe form of the disease and the most common — under Biogen’s expanded access program.
The ministry signaled in January that it was likely to approve Spinraza this year when it designated nusinersen an orphan drug, a declaration that opened the door to accelerated approval. Normal approval can take a year or more.
Although the patient registry started in 2016 by SMA Family Foundation Russia now contains 800 names, the organization believes that, based on Russia’s population of 147 million, up to 3,000 people have the disease.
Besides the 40 children receiving Spinraza via expanded access, about 20 Russians are getting it in Europe, where their families moved to obtain access. The European Medicines Agency (EMA) authorized Spinraza in June 2017, six months after the U.S. Food and Drug Administration (FDA) became the first agency to grant approval.
While EMA approval made Spinraza theoretically available to all SMA patients in the 28-nation European Union simultaneously, each EU country then had to decide whether its health insurance system would cover the treatment — a process that requires negotiating prices with Biogen individually. All 28 countries except Estonia and Latvia now have reached such agreements with the company.
Eight EU countries — Austria, France, Germany, Italy, Luxembourg, Poland, Portugal and Romania — reimburse for all SMA patients regardless of age, disease severity or any other criteria.
The SMA Family Foundation Russia, whose membership has grown from eight families when it started in October 2014 to more than 800 today, lobbied hard for Health Ministry approval of Spinraza.
Germanenko said her organization won’t rest until Russia’s health insurance system covers the treatment.
“From the time Spinraza was approved at the end of 2016 until now, Russia has lost about 100 SMA patients,” she said, acknowledging that obtaining such coverage from Russia’s cash-strapped healthcare system will be an uphill battle due to Spinraza’s extremely high cost.
Five Russian clinics now administer Spinraza under the expanded access program — three in Moscow, one in Yekaterinburg near the Ural Mountains, and one in the Siberian city of Barnaul. Treatment is limited to type 1 children 3 years and younger who do not require ventilators, Germanenko said.
Besides pushing for expanded access, the foundation has helped establish a network of eight Russian clinical trial facilities, Germanenko said. These have been used by Swiss drugmakers Roche, to test risdiplam, and Novartis, to test branaplam. The risdiplam trials started in mid-2018 and continue. The branaplam trials — part of a multi-country testing program — ran from October 2018 to March 2019.
The foundation also has asked Novartis to test its SMA gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in Russia. In May, the FDA became the world’s first regulatory authority to approve Zolgensma.