RIYADH: The Saudi Food and Drug Authority has approved the registration of Qalsody (Tofersen) for the treatment of adults with amyotrophic lateral sclerosis linked to mutations in the SOD1 gene, the Saudi Press Agency reported.
ALS is a rare neurodegenerative disease that affects the nerve cells responsible for voluntary movement, gradually causing muscle weakness and leading to loss of mobility over time.
According to the SPA, the approval is part of the SFDA’s efforts to facilitate patient access to treatment, as Qalsody has been designated an orphan drug for the treatment of rare diseases.
Normally, the SOD1 protein helps eliminate toxic byproducts produced during regular cellular activity.
In cases where a genetic mutation in the SOD1 gene causes ALS, a defective protein that fails to protect the cell from toxins is produced. The faulty protein accumulates within the cell, disrupting normal cellular processes.
