SFDA Fast-Tracks Access to Qalsody Under the Orphan Drug Program

The Saudi Food and Drug Authority (SFDA) has approved the registration of Qalsody (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) linked with mutations in the superoxide dismutase 1 (SOD1) gene. ALS is a rare and progressive motor neuron disease that disturbs the nerve cells controlling voluntary movement, leading to increasing muscle weakness and loss of mobility. The approval underscores the SFDA’s ongoing commitment to expanding access to therapies for rare diseases, with Qalsody granted Orphan Drug designation.

Qalsody has got a unique mechanism of action that works by an antisense oligonucleotide approach, designed to target and reduce the faulty SOD1 protein production at its genetic source. By binding to messenger RNA (mRNA), it helps slow the harmful buildup that contributes to nerve damage.

Data supporting the approval showed a reduction in neurofilament light chain (NfL) levels an indicator of slower neurodegeneration and lower SOD1 protein concentrations in the cerebrospinal fluid. These findings highlight Qalsody’s potential to modify disease progression, though long-term benefits continue to be studied.

The most common side effects observed included muscle and joint pain, fatigue, injection-site pain, fever, and elevated cerebrospinal fluid protein.

This breakthrough reflects the SFDA’s ongoing dedication to advancing healthcare innovation and aligns with the goals of Saudi Vision 2030 ensuring patients have faster access to effective, high-quality treatments that transform lives.

Learn more: GovSFDA Approves the Registration of Qalsody for the Treatment of Amyotrophic Lateral Sclerosis (ALS)