Chiesi Global Rare Diseases Announces FDA Approval of Juxtapid (lomitapide) Capsules for Pediatric Use in Homozygous Familial Hypercholesterolemia (HoFH)

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BOSTON, March 03, 2026 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, announced today that the U.S. Food and Drug Administration (FDA) has approved Juxtapid (lomitapide) capsules for pediatric use in homozygous familial hypercholesterolemia (HoFH) for children 2 years of age and older. Juxtapid is a proven therapy that has been approved in the United States for adult patients with HoFH to be used along with diet and other lipid-lowering treatments, since 2012.

  • Approval expands indication of Juxtapid to include children 2 years of age and older with HoFH
  • This announcement was made during the week of Rare Disease Day, which aims to raise awareness about the impact of rare diseases on patients

    • HoFH, an ultra-rare genetic disorder, impacts an estimated 1 in 250,000 to 1 in 360,000 individuals worldwide, including children, who can be born with dangerously high low-density lipoprotein cholesterol (LDL-C) levels.1,2 Without early intervention, those living with this condition face a significantly increased risk of severe atherosclerosis.3,4

    “This approval represents more than a regulatory milestone; it’s a meaningful advancement for children and families living with HoFH,” said Mitch Goldman, Senior Vice President, R&D, Chiesi Global Rare Diseases. “By expanding access to Juxtapid for children 2 years of age and older, we’re enabling very young members of the HoFH community to benefit from the same proven treatment that has already helped adults manage their condition. This achievement reflects our unwavering commitment to bringing safe, effective therapies to those who need them most and to making a lasting difference at every stage of life.”

    “Children with HoFH face extraordinary challenges from the moment they’re diagnosed,” said Katherine Wilemon, Founder and CEO of Family Heart Foundation. “Their lives are shaped by frequent medical visits and the constant worry of cardiovascular risk at an age when most kids are just learning to ride a bike or play sports. The recent treatment approval for this age group marks a meaningful step forward for young children impacted by HoFH.”

    The FDA’s approval is grounded on evidence from a Phase 3, open-label, multicenter study (APH-19) evaluating the safety and efficacy of Juxtapid® in 43 pediatric participants aged 5 to 17 years with HoFH who were receiving standard-of-care lipid-lowering therapy and a low-fat diet.5 Over a 24-week treatment period, Juxtapid was titrated to each participant’s highest tolerated dose. The study demonstrated a mean 49% reduction in low-density lipoprotein cholesterol (LDL-C) from baseline, accompanied by significant decreases in non-high-density lipoprotein cholesterol (non-HDL-C), total cholesterol, very low-density lipoprotein cholesterol (VLDL-C), apolipoprotein B, and triglycerides. Adverse events were primarily gastrointestinal or hepatic in nature, consistent with the known safety profile of Juxtapid. An integrated model-based analysis confirmed that Juxtapid is safe and efficacious for the treatment of HoFH in pediatric populations and support the dose justification in children > 2 yrs old. Overall, the results showed that Juxtapid achieved substantial, clinically meaningful reductions in LDL-C in pediatric use for HoFH.6

    USE and IMPORTANT SAFETY INFORMATIONJuxtapid (lomitapide) is a prescription medicine used along with a low-fat diet, exercise and other low-density lipoprotein (LDL) lowering medicines to reduce LDL-C in adults and children 2 years of age and older with a type of high cholesterol called homozygous familial hypercholesterolemia (HoFH).

    It is not known if Juxtapid is safe and effective in people with kidney problems including people with end-stage kidney disease who are not on dialysis.

    It is not known if Juxtapid is safe and effective when used in children under the age of 2.

    IMPORTANT SAFETY INFORMATION

    What is the most important information I should know about Juxtapid?

  • Juxtapid is available only through certified pharmacies that are enrolled in the Juxtapid REMS Program. Your healthcare provider must be enrolled in the program in order for you to be prescribed Juxtapid.
  • There is a registry that collects information about the effects of taking Juxtapid over time. Ask your healthcare provider for more information about this registry or visit www.Juxtapid.com or call 1-877-902-4099.

    • Juxtapid may cause serious side effects, including:

    Liver Problems:

  • Juxtapid can cause liver problems such as increased liver enzymes or increased fat in the liver. For this reason, your healthcare provider should do blood tests to check your liver before you start Juxtapid, if your dose is increased, and while you take Juxtapid. If your tests show signs of liver problems, your doctor may lower your dose of Juxtapid or stop it altogether.
  • You should tell your doctor if you have had liver problems in the past, including liver problems while taking other medicines.
  • Juxtapid may cause nausea, vomiting and stomach pain, especially if you do not eat a low-fat diet. These side effects can also be symptoms of liver problems. Tell your healthcare provider right away if you have nausea; vomiting or stomach pain that gets worse, does not go away, or changes; fever; flu-like symptoms; yellowing of your eyes or skin; or are feeling more tired than usual while taking Juxtapid.
  • Do not drink more than 1 alcoholic drink per day while taking Juxtapid.

    • Harm to your unborn baby:

  • Do not take Juxtapid if you are pregnant, think you may be pregnant, or are planning to become pregnant.
  • If you are a female who can get pregnant, you should have a negative pregnancy test result before you start on Juxtapid. Use effective birth control while taking Juxtapid and for 2 weeks after your last dose of Juxtapid. If you become pregnant while taking Juxtapid, stop taking Juxtapid and call your healthcare provider right away.

    • Do not take Juxtapid if you:

  • Are taking medicines that are strong or moderate CYP34A inhibitors (for example, certain medicines used to treat bacterial, fungal, or viral infections, and medicines used to treat depression, high blood pressure or chest pain). These medicines may affect how your body breaks down Juxtapid.
  • Drink grapefruit juice.
  • Have moderate to severe liver problems or active liver disease, including abnormal liver function tests.

    • Before you take Juxtapid, tell your healthcare provider about all of your medical conditions, including if you have liver problems; have kidney problems; have intestine or bowel problems; drink alcohol; are breastfeeding or plan to breastfeed. It is not known if Juxtapid passes into your breastmilk. You and your healthcare provider should decide if you will take Juxtapid or breastfeed. You should not do both.

    Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Juxtapid may affect the way other medicines work, and other medicines may affect how Juxtapid works.

    Other possible side effects of Juxtapid include:

  • Problems absorbing certain nutrients. Juxtapid makes it harder for some fat-soluble nutrients, such as vitamin E and fatty acids, to get into your body. Take supplements that contain fat-soluble vitamins each day while you take Juxtapid.
  • Gastrointestinal symptoms. Diarrhea, nausea, vomiting, and stomach pain or discomfort are very common when taking Juxtapid. Strictly following a low-fat diet may help lower the chance of having these symptoms. Stop taking Juxtapid and tell your healthcare provider if you have severe diarrhea, especially if you also have lightheadedness, decreased urine output, or tiredness.
  • Muscle pain, tenderness and weakness (myopathy). This can happen when Juxtapid is taken with certain doses of simvastatin and lovastatin. Tell your doctor right away if you have unexplained muscle pain, tenderness, or weakness, especially if you have a fever or feel more tired than usual while taking Juxtapid.
  • Increased levels of certain blood thinners. Juxtapid can increase the level of the blood thinner, warfarin. If you take warfarin, your healthcare provider should check your blood clotting times frequently, especially after your dose of Juxtapid changes.
  • Liver problems caused by certain drugs. Certain medicines can cause liver problems, including isotretinoin, acetaminophen, methotrexate, tetracyclines, and tamoxifen. If you take these medicines with Juxtapid your healthcare provider may do blood tests more often to check your liver.
  • The most common side effects of Juxtapid in adults and children 5 years of age and older include: diarrhea, nausea, stomach (abdominal) pain, indigestion, vomiting, and increases in liver enzymes.

    • Tell your healthcare provider if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of Juxtapid. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects.

    You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.FDA.gov/medwatch or call 1-800-FDA-1088.

    About HoFHHomozygous familial hypercholesterolemia (HoFH) is a serious, rare genetic disease that impairs the function of the receptor responsible for removing low-density lipoprotein cholesterol (LDL-C) ("bad" cholesterol) from the body. A loss of low-density lipoprotein (LDL) receptor function results in extreme elevation of blood cholesterol levels. Individuals with HoFH often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.

    About Juxtapid® (lomitapide)Juxtapid (lomitapide) is a prescription medicine used along with a low-fat diet, exercise and other low-density lipoprotein (LDL) lowering medicines to reduce LDL-C in adults and children 2 years of age and older with a type of high cholesterol called homozygous familial hypercholesterolemia (HoFH).

    About Chiesi GroupChiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.

    By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.

    With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.

    For more information visit www.chiesi.com.

    About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system.

    For more information visit www.chiesirarediseases.com.

    References

  • Cuchel, M., Raal, F. J., Hegele, R. A., Al-Rasadi, K., Arca, M., Averna, M., Bruckert, E., Freiberger, T., Gaudet, D., Harada-Shiba, M., Hudgins, L. C., Kayikcioglu, M., Masana, L., Parhofer, K. G., Roeters van Lennep, J. E., Santos, R. D., Stroes, E. S. G., Watts, G. F., Wiegman, A., Stock, J. K., … Ray, K. K. (2023). 2023 Update on European Atherosclerosis Society Consensus Statement on Homozygous Familial Hypercholesterolaemia: new treatments and clinical guidance. European heart journal, 44(25), 2277–2291.
  • Mainieri, F., Tagi, V. M., & Chiarelli, F. (2022). Recent Advances on Familial Hypercholesterolemia in Children and Adolescents. Biomedicines, 10(5), 1043.
  • Graves, L. E., Horton, A., Alexander, I. E., & Srinivasan, S. (2023). Gene Therapy for Paediatric Homozygous Familial Hypercholesterolaemia. Heart, Lung and Circulation, 32(7), 769-779.
  • Nohara, A., Tada, H., Ogura, M., Okazaki, S., Ono, K., Shimano, H., Daida, H., Dobashi, K., Hayashi, T., Hori, M., Matsuki, K., Minamino, T., Yokoyama, S., & Harada-Shiba, M. (2021). Homozygous Familial Hypercholesterolemia. Journal of Atherosclerosis and Thrombosis, 28(7), 665.
  • Masana L, Zambon A, Schmitt CP, Taylan C, Driemeyer J, Cohen H, Buonuomo PS, Alashwal A, Al-Dubayee M, Kholaif N, Diaz-Diaz JL, Maatouk F, Martinez-Hervas S, Mangal B, Löwe S, Cunningham T. Lomitapide for the treatment of paediatric patients with homozygous familial hypercholesterolaemia (APH-19): results from the efficacy phase of an open-label, multicentre, phase 3 study. Lancet Diabetes Endocrinol. 2024 Dec;12(12):880-889. doi: 10.1016/S2213-8587(24)00233-X. Epub 2024 Oct 16. PMID: 39426393.
  • Juxtapid (lomitapide) Prescribing Information. Chiesi Farmaceutici S.p.A.; 2026

    • Source: Chiesi Global Rare Diseases

    Source: HealthDay

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    Posted : 2026-03-04 08:45

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