In May this year, the European Commission (EC) granted marketing authorisation for the active substance Nusinersen to treat 5q spinal muscular atrophy (SMA) and for the active substance Cerliponase alfa to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease.
Until now there was no approved treatment either for SMA nor for CLN2 in the European Union. The EC based its green light for the two substances on positive opinions issued by the European Medicines Agency, EMA, earlier this year.
Each of the 28 EU Member States will now decide on their own price and reimbursement policy and consider the potential role/use of the medicines in the context of their national health systems. In Germany for example, the statutory health insurance will bear the costs of the treatment as soon as the substances are available on the market.
Nusinersen was approved by the U.S. Food and Drug Administration in December of 2016 and Cerliponase alfa in April of 2017.
According to the European Medicines Agency, Cerliponase alfa is expected to improve some of the symptoms of CLN2: In a study, 20 of the 24 patients (ages three to eight years) treated with the substance experienced a slower progression of the disease than expected, a stabilisation of the progression or some improvement of their motor and language abilities.
The number of patients in the study treated with Cerliponase alfa was very limited because CLN2 is an extremely rare genetic disease which affects approximately less than 2000 children worldwide. During the treatment with Cerliponase alfa, fever, vomiting, hypersensitivity, seizures and upper respiratory tract infections were the most common adverse events.
According to EMA, 51% of those patients treated with Nusinersen showed a pre-defined improvement of their motor skills (e.g. head control, sitting, crawling, standing, walking). The remaining 49% of the patients were considered as non-responders to the treatment. During the clinical trials with Nusinersen, the most frequent side effects were constipation as well as upper and lower respiratory infection.
Currently, little is known about the positive effects of Nusinersen and Cerliponase alfa in the long term – but according to EMA, more information will become available with time.
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Sabine Kraft is Chair of the Board of Trustees of the ICPCN and CEO of „Bundesverband Kinderhospiz e.V.“, the national association of children’s hospices in Germany.