SFDA Approves Qalsody For Treating ALS Linked To SOD1 Mutations

The Saudi Food and Drug Authority (SFDA) has approved Qalsody (tofersen) for adults with amyotrophic lateral sclerosis (ALS) linked to a mutation in the superoxide dismutase 1 (SOD1) gene. ALS is a rare disorder affecting motor neurons, leading to muscle weakness and loss of mobility. This approval is part of SFDA's ongoing efforts to improve access to treatments for rare diseases.

Qalsody is part of the Orphan Drug Program, which aims to expedite access to promising therapies for rare conditions. The drug uses an antisense oligonucleotide approach, where a synthetic nucleotide strand binds to messenger RNA (mRNA). This binding reduces the production of the targeted protein, decreasing its accumulation in the body.

The approval followed a thorough evaluation of Qalsody's efficacy, safety, and quality. Clinical trials showed reduced neurofilament light chain (NfL) levels compared to placebo, indicating potential slower neurodegeneration. Additionally, there was a decrease in SOD1 protein concentrations in cerebrospinal fluid, suggesting effective molecular target engagement.

Ongoing Research and Safety Concerns

SFDA noted that while biomarkers are promising, they cannot replace proven clinical outcomes. Long-term benefits are still being studied in ongoing trials. Common side effects reported during studies included muscle pain, joint pain, fatigue, injection-site discomfort, fever, and elevated cerebrospinal fluid protein levels.

"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 treats conditions affecting fewer than five individuals per 10,000 people in Saudi Arabia. For more details on the Orphan Drugs Guideline or inquiries, visit SFDA's website or contact them via email at [email protected].

With inputs from SPA