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(11) Alternative drugs approved for homozygous familial hypercholesterolemia treatment, including a microsomal triglyceride transfer protein inhibitor (lomitapide) and an antisense RNA compound (mipomersen), may be feasible for treating FCS.
Novel agents include the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors (that have shown to lead to a reduction in CVD morbidity), mipomersen, cholesterol ester transfer protein (CETP) inhibitors and bempedoic acid.
Although there are no data available for children, oral lomitapide and injectable mipomersen have recently been approved in the United States (US) as adjunct therapies for HoFH in patients aged [greater than or equal to]18 and [greater than or equal to]12 years, respectively (1).
Access to inhibitors of PCSK9 and more costly medications, such as mipomersen or lomitapide, may depend on a diagnosis of FH in some countries (25-28).
Mipomersen (inhibitor of apolipoprotein B-100), an antisense oligonucleotide complementary to the coding region of human apo-B mRNA, was approved by the FDA in January 2013.
(18, 19) In addition, injectable mipomersen, an antisense RNA therapy is approved in the USA from 12 years old and can reduce LDL-C up to 25%.
No reports describing the use of mipomersen (Kynamro), an oligonucleotide inhibitor of apolipoprotein B-100 synthesis, in human pregnancy have been located.
* Kynamro (mipomersen; Sanofi): What factors will determine whether Kynamro reaches the European market?
Kastle Therapeutics, a biopharmaceutical company, has signed a contract to acquire global rights to develop and commercialise Kynamro (mipomersen sodium) injection from Ionis Pharmaceuticals.
US-based drugmaker Kastle Therapeutics, LLC has acquired global rights to develop and commercialize Kynamro (mipomersen sodium) injection from Ionis Pharmaceuticals, Inc.
(22) For patients with HoFH, in whom the condition is more quickly life-threatening, there are additional choices, including LDL apheresis and medications such as mipomersen and lomitapide.
The potential future therapeutic options for targeted Lp(a) control include cholesterol-ester transfer protein inhibitors (CETPI), anti-sense oligonucleopeptides (ASO) and proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK-9I) and L-carnitine.22,23 The CETP inhibitor Anacetrapib has been shown to reduce Lp(a) plasma levels by up to 50%.24 The ASO Mipomersen lowers Lp(a) by up to 50% in phase 2 trials with single injections given fortnightly.25 Maximum reductions occur after 3-6 months of continuous therapy, and slowly return to baseline after cessation of therapy.25 The PCSK-9I, AMG-145, has similar effects on Lp(a) levels with once monthly injections.26 Carnitine supplementation therapy in doses of up to 1gm daily leads to 10-15% reduction (Table-2).23,24