The Expert Council of the Circle of Good Foundation has expanded the age limits for the use of therapies for rare diseases: spinal muscular atrophy and cystic fibrosis. This decision was the response of the expert community to the appeal of Aleksandr Tkachenko, the Chairman of the Board of the Foundation, in connection with the forthcoming registration of Zolgensma in Russia.
In September, Tkachenko appealed to the medical community with a request to analyze the existing medical experience of the use of Zolgensma. Aleksander Tkachenko noted that since the release of the drug on the market, significant experience has been accumulated both in Russia and abroad, which makes it possible to adjust the approaches to the use of the drug in the treatment of children with SMA.
On December 9, 2021, Zolgensma was registered in the Russian Federation. The experts’ decision to expand the age limits takes into account manufacturer’s instructions for Zolgensma, which were approved as part of the registration of the drug. This means it is now possible to provide the drug to newly identified children with SMA who have not received any pathogenetic therapy, as well as to those who have received other therapies.
The Expert Council also once again stated that it is impossible to use other types of pathogenetic therapy after the use of Zolgensma until the safety and, most importantly, the effectiveness of combinations of different types of SMA therapies have been confirmed during clinical trials.
Today the Foundation, satisfying all applications it has received, provides medicines to 1076 children diagnosed with SMA, including 21 children who reveived Zolgensma at the expense of the fund. One package costs over 100 million rubles.
Also at the meeting of the expert council on December 9, it was decided to expand the age limits for the use of Trikafta, a therapy for cystic fibrosis. From 2022, the Circle of Kindness will be able to purchase Trikafta for those who have medical indications and the decision of the federal case conference for all patients with cystic fibrosis aged six to 18 years. Earlier restrictions related to the severity of the disease were also lifted.