Treatment with Nusinersen is available for people with 5q-associated spinal muscular atrophy (5q-SMA). Approval of Nusinersen is based on clinical trials conducted in children with 5q-SMA. Adult SMA patients were not included in the pivotal studies. Despite the limited studies, adult 5q-SMA patients are also treated with Nusinersen, as the same disease mechanism is assumed in children and adults.
With this in mind, APST is conducting a study in people with 5q-SMA receiving Nusinersen therapy in adulthood. This will systematically examine treatment goals, symptom progression, treatment satisfaction, and likelihood to recommend the drug to others.
The study started in July 2019 and will run until June 2021, with initial results presented at the German Society of Neurology Congress in November 2020.
To date (as of 11 2020), 151 adults with 5q-SMA have been enrolled. Results to date show that in adult SMA patients, Nusinersen is used across a broad age spectrum. Treatment initiation occurred with high variability in motor-functional deficits. The treatment goals of Nusinersen therapy are individualized and relate to specific target symptoms (e.g., preservation of hand function). Improvement of target symptoms was achieved in the majority of patients. Global therapy satisfaction is high, although the user-friendliness of the therapy is rated more critically. Satisfaction with Nusinersen - determined with the probability of recommendation for the drug - is exceptionally high.
Overall, the results of the use study show a positive perception of Nusinersen therapy among SMA patients who received treatment with Nusinersen in adulthood.
The results of the study to date will soon be published in an international neurological journal.
Download leaflet (PDF, in German) on the Nusinersen application study
Expectation horizon for Nusinersen therapy in adults with SMA