SFDA Approves Lojuxta For Pediatric Treatment Of Homozygous Familial Hypercholesterolemia

The Saudi Food and Drug Authority (SFDA) has approved the use of Lojuxta (lomitapide) for children aged five and older with Homozygous Familial Hypercholesterolemia (HoFH). This makes SFDA the first global regulatory body to approve Lojuxta for pediatric use, showcasing its leadership in providing advanced treatments for rare diseases.

Lojuxta functions by inhibiting the Microsomal Triglyceride Transfer Protein (MTP), which is crucial in forming Apo B lipoproteins in the liver and intestines. By blocking MTP, Lojuxta reduces lipoprotein secretion and lowers circulating cholesterol and triglycerides. It is used alongside a low-fat diet and other lipid-lowering medications, with or without LDL apheresis.

Clinical trials have shown that Lojuxta can lower LDL-C levels by over 50% in children with HoFH after 24 weeks of treatment. These results also indicated improvements in patients' overall lipid profiles, suggesting a potential reduction in cardiovascular risks associated with this genetic disorder.

The SFDA's approval followed a thorough evaluation of Lojuxta's safety and efficacy based on established regulatory standards. The decision underscores SFDA’s dedication to fostering innovation and expanding access to cutting-edge treatments, aligning with Saudi Vision 2030’s Health Sector Transformation Program.

Common side effects reported include gastrointestinal issues such as diarrhea, nausea, and abdominal pain. Patients may also experience elevated liver enzymes and hepatobiliary disorders like hepatic steatosis, hepatomegaly, and hepatotoxicity. Regular liver function monitoring is advised during treatment due to these risks.

SFDA stresses the importance of confirming HoFH genetically when possible. Other primary hyperlipoproteinemia forms and secondary hypercholesterolemia causes, like nephrotic syndrome or hypothyroidism, should be ruled out before starting treatment.

This approval signifies SFDA’s ongoing commitment to improving healthcare quality by enhancing access to advanced therapies for rare conditions. It reflects their role in advancing medical innovation within the framework of Saudi Vision 2030's health initiatives.

With inputs from SPA