Riyadh: The Saudi Food and Drug Authority (SFDA) has approved the registration of Qalsody (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene. ALS is a rare motor neuron disorder that progressively affects the nerve cells responsible for voluntary movement, leading to muscle weakness, and eventually, a loss of mobility.
According to Saudi Press Agency, this approval reflects SFDA’s continued efforts to facilitate access to treatments for rare diseases, with Qalsody designated under the Orphan Drug Program.
Qalsody works through an antisense oligonucleotide approach. A short synthetic strand of nucleotides is designed to bind complementarily to the messenger RNA (mRNA), thereby limiting its ability to direct the production of the targeted protein. This process reduces the production and accumulation of the protein in the body.
Approval of Qalsody is based on a comprehensive assessment of the totality of evidence, including its efficacy, safety, and quality, in line with established regulatory requirements. Clinical trial results showed a reduction in neurofilament light chain (NfL) levels versus placebo, a biomarker potentially associated with slower neurodegeneration. The studies also showed a decrease in SOD1 protein concentrations in cerebrospinal fluid, indicating molecular-level target engagement.
SFDA emphasized that confirmation of long-term clinical benefit remains under investigation in ongoing trials, and biomarkers cannot substitute for proven clinical outcomes.
The most commonly reported adverse events during clinical studies included myalgia, arthralgia, fatigue, injection-site pain, fever, and elevation of cerebrospinal fluid protein.
“This approval highlights SFDA’s commitment to enhancing access to treatments for rare and hard-to-treat diseases through the Orphan Drug Program, which aims to accelerate access to promising therapies and address unmet medical needs, in alignment with the objectives of the Healthcare Sector Transformation Program under Saudi Vision 2030 to improve the quality of healthcare services,” said the release.
An orphan drug is defined as a medication intended to treat a rare disease or condition that affects fewer than five individuals per 10,000 people in the Kingdom of Saudi Arabia.
