The Saudi Food and Drug Authority (SFDA) has approved a new indication for Lojuxta (lomitapide), authorizing its use in children aged five years and older diagnosed with Homozygous Familial Hypercholesterolemia (HoFH). With this decision, the SFDA becomes the first regulatory body worldwide to permit pediatric use of Lojuxta, underscoring its leadership in expanding access to advanced therapies for rare diseases.
According to a press release issued today, Lojuxta functions by inhibiting Microsomal Triglyceride Transfer Protein (MTP), a key component involved in forming Apo B–containing lipoproteins in the liver and intestines. By blocking MTP, the drug decreases lipoprotein secretion and lowers circulating levels of lipoprotein-bound cholesterol and triglycerides.
Lojuxta
Lojuxta is approved as an adjunct to a low-fat diet and other lipid-lowering treatments, and it can be used with or without low-density lipoprotein (LDL) apheresis. The SFDA also recommends obtaining genetic confirmation of HoFH whenever possible. It further emphasized the need to rule out other types of primary hyperlipoproteinemia and secondary causes of hypercholesterolemia, such as nephrotic syndrome or hypothyroidism.
The pediatric approval follows a comprehensive evaluation of Lojuxta’s safety and effectiveness. Clinical studies showed that the medication reduced LDL-C levels by more than 50% in children with HoFH after 24 weeks, along with overall improvements in lipid profiles. These findings highlight Lojuxta’s potential to reduce the risk of severe cardiovascular complications associated with this genetic condition.
Reported side effects most commonly included gastrointestinal symptoms such as diarrhea, nausea, and abdominal pain. Some patients also experienced elevated liver enzymes and hepatobiliary disorders—including hepatic steatosis, hepatomegaly, and hepatotoxicity—prompting recommendations for regular liver-function monitoring during treatment.
This approval reflects the SFDA’s ongoing commitment to fostering innovation and expanding access to advanced therapies, in line with the objectives of the Health Sector Transformation Program, a key pillar of Saudi Vision 2030.
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