Riyadh, Saudi Arabia – December 1, 2025
The Saudi Food and Drug Authority (SFDA) has achieved a major regulatory milestone by approving a new pediatric indication for Lojuxta (lomitapide), marketed by Chiesi Farmaceutici. The drug is now authorized for the treatment of children five years older diagnosed with homozygous familial hypercholesterolemia (HoFH), a rare and severe genetic cholesterol disorder. This approval makes the SFDA the first regulatory authority in the world to authorize Lojuxta for this age group.
SFDA confirmed that the decision was based on a rigorous assessment of clinical data demonstrating that Lojuxta reduced LDL-C levels by more than 50% after 24 weeks in pediatric patients. The medication also improved overall lipid profiles and is used together with a low-fat diet and other lipid-lowering therapies, with or without LDL-C apheresis. The Authority emphasized the importance of genetic testing to confirm HoFH and rule out secondary causes.
This landmark approval supports the goals of the Health Sector Transformation Program under Saudi Vision 2030 and reflects continued enhancements in SFDA drug registration standards. By authorizing the first pediatric use worldwide, the SFDA reinforces its leadership in providing advanced treatment options for patients with rare diseases.
The regulatory support in Saudi Arabia was provided by PharmaKnowl, a Saudi regulatory consulting firm, whose expertise in SFDA regulatory pathways and clinical evaluation played a central role in successfully securing this first-in-the-world pediatric authorization.