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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.

The SFDA said that Qalsody works to reduce the production of the defective SOD1 protein through a novel therapeutic approach known as antisense therapy. This technique employs small molecules called nucleotides, designed to bind precisely to a specific sequence of mRNA generated by the genetic mutation that causes the protein to misfold. This process limits the cell’s ability to produce the defective protein, reducing its accumulation in the body.

The authority added that the drug’s approval followed a thorough assessment of its efficacy, safety and quality. Clinical studies of patients who received the drug showed a reduction in key indicators of nerve damage, most notably neurofilament light levels, compared with patients who received a placebo. A decrease was also observed in the concentration of the defective SOD1 protein in cerebrospinal fluid, confirming that the drug is acting on its intended molecular target. The authority said, however, that the long-term benefits are still under evaluation and that these early findings are not a substitute for confirmed clinical outcomes.

The SFDA reported that the most commonly observed side effects in clinical studies were muscle and joint pain, fatigue, injection site discomfort, fever and increased levels of certain proteins in the cerebrospinal fluid.

The approval reflects the SFDA’s commitment to expanding access to effective treatments for rare and hard-to-treat diseases through the Orphan Drugs Program, a key strategic initiative designed to accelerate the availability of promising therapies and address unmet medical needs.

It aligns with the goals of the Health Sector Transformation Program, one of the key initiatives of the Kingdom’s Vision 2030, which seeks to improve the quality of healthcare services.

An orphan drug is defined as a medication developed to treat rare diseases that affect fewer than five in 10,000 people in the Kingdom.

The orphan drugs guide is available on the authority’s website at https://www.sfda.gov.sa/ar/regulations/88482. For further inquiries, contact the SFDA via email at [email protected].