Riyadh: The Saudi Food and Drug Authority (SFDA) announced its approval of a new medical claim for Lojuxta (lomitapide), allowing its use to treat children aged five years and older diagnosed with Homozygous Familial Hypercholesterolemia (HoFH). This approval positions SFDA as the first regulatory authority worldwide to authorize the pediatric use of Lojuxta, highlighting its global leadership in improving access to advanced therapies for rare disorders.
According to Saudi Press Agency, SFDA indicated that Lojuxta works by inhibiting the Microsomal Triglyceride Transfer Protein (MTP). MTP plays a key role in the assembly of Apo B containing lipoproteins in the liver and intestines. Inhibition of MTP reduces lipoprotein secretion and circulating concentrations of lipoprotein-borne lipids including cholesterol and triglycerides.
Lojuxta is indicated as an adjunct therapy to a low-fat diet and other lipid-lowering medications, and may be used with or without low density lipoprotein (LDL) apheresis. SFDA also emphasizes that genetic confirmation of HoFH should be obtained whenever possible.
The release added that other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolaemia (e.g., nephrotic syndrome, hypothyroidism) must be excluded.
SFDA explained that the pediatric approval of Lojuxta was granted following a comprehensive assessment of its efficacy and safety in accordance with established regulatory standards. Clinical studies demonstrated that Lojuxta achieved a reduction in LDL-C levels exceeding 50% in children patients with HoFH after 24 weeks of treatment. This significant result was accompanied by overall improvements in patients’ lipid profiles. These positive findings strongly suggest the drug’s potential to help reduce the risk of serious cardiovascular complications in individuals affected with this genetic disorder.
Clinical data indicated that the most frequently reported side effects included gastrointestinal disturbances, such as diarrhea, nausea, and abdominal pain. Additionally, patients may experience elevated liver enzymes and hepatobiliary disorders, including hepatic steatosis, hepatomegaly, and hepatotoxicity. In light of these risks, regular monitoring of liver function is recommended throughout the treatment period.
This approval reflects the SFDA’s continued commitment to advancing innovation, expanding access to advanced treatment options, thereby enhancing the quality of healthcare in alignment with the goals of the Health Sector Transformation Program, one of the key initiatives of Saudi Vision 2030.
