Global Leadership for Increasing Patient Accessibility, SFDA Expands Lojuxta Indication to Include Treatment of Children with Homozygous Familial Hypercholesterolemia (HoFH)
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 5 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.
Mechanism of Action of Lojuxta
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. 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.
){kind=link}