Posted by Michael Wonder on 08 Nov 2021
Drugs against rare diseases: Libmeldy gene therapy shows additional benefits - Zolgensma cell therapy, on the other hand, does not
4 November 2021 - The Federal Joint Committee (G-BA) today assessed the additional benefit of two active ingredients against very rare, serious, genetic diseases in children.
The G-BA sees a considerable additional benefit in the Libmeldy cell therapy, which is used against the metabolic disorder metachromatic leukodystrophy in sick but still symptom-free children.
This is different with the Zolgensma gene therapy, which is approved against spinal muscular atrophy. On the basis of the available data, the G-BA did not find any additional benefit compared to the comparator therapy for any patient group.
