What is Lojuxta - lomitapide used for and what is it used for?
Lojuxta is a medicine that contains the active substance lomitapide, used to treat adult patients with homozygous familial hypercholesterolemia, an inherited disorder that causes blood cholesterol levels (a type of fat) to rise. The medicine is used in combination with a low-fat diet and other medicines to reduce the level of fat in the blood. When possible, the patient's illness must be diagnosed by genetic testing.
How is Lojuxta used - lomitapide?
Lojuxta can only be obtained with a prescription. It is available as capsules (5, 10 and 20 mg) to be taken by mouth on an empty stomach, at least two hours after the evening meal. Therapy must be started and monitored by a doctor experienced in treating conditions that cause an increase in blood fat levels. Treatment should start at a dose of 5 mg once a day; if well tolerated, the dosage can be gradually increased to reach the maximum dose of 60 mg. Patients with reduced liver function or patients on kidney dialysis and those taking certain other medicines will need to take a reduced dose. For more information, see the package leaflet.
How does Lojuxta - lomitapide work?
The active ingredient in Lojuxta, lomitapide, blocks the action of a substance in the body called the "microsomal triglyceride transfer protein", which is found in liver cells and in the intestine. This substance is responsible for the accumulation of fatty substances such as cholesterol and triglycerides in larger particles called lipoproteins, which are subsequently released into the blood. By blocking this protein, Lojuxta decreases the level of fats released into the blood, thus helping to reduce the cholesterol level in hypercholesterolemia.
What benefit has Lojuxta - Lomitapide shown during the studies?
The benefits of Lojuxta in the reduction of blood cholesterol were evaluated in a main study involving 29 patients with homozygous familial hypercholesterolemia. All patients were treated with Lojuxta in combination with other medicines to reduce the level of fat in the blood. Lojuxta has not been compared with other therapies. The main measure of effectiveness was the change in blood levels of LDL cholesterol (low density lipoprotein), commonly known as "bad cholesterol", in patients 26 weeks after treatment. On average, LDL cholesterol levels in patients have been reduced by 40%.
What is the risk associated with Lojuxta - lomitapide?
The most serious side effect seen in some patients treated with Lojuxta is an abnormal increase in liver enzyme levels. The most common side effects are intestinal problems, which can affect up to 9 out of 10 people: diarrhea, nausea, dyspepsia (heartburn) and vomiting have been observed in more than 3 in 10 people, while at least 2 in 10 reported pain, discomfort and abdominal bloating, constipation and flatulence. For the full list of all side effects reported with Lojuxta, see the package leaflet. Lojuxta should not be used in pregnant women. Furthermore, it must not be used in patients with moderate to severe hepatic insufficiency or with liver test results that are out of the ordinary and unexplained, or in patients with significant or long-term intestinal problems. Lojuxta should not be used concomitantly with a dose of simvastatin (another medicine used to lower blood cholesterol levels) greater than 40 mg or with certain other medicines that affect the breakdown of lomitapide in the body. For the full list of limitations, see the package leaflet.
Why has Lojuxta - lomitapide been approved?
The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Lojuxta's benefits are greater than its risks and recommended that it be approved for use in the EU. The CHMP took into account that patients with homozygous familial hypercholesterolaemia with an unmet medical demand have found benefit in terms of reducing LDL cholesterol levels. However, the CHMP noted that the long-term benefit to the cardiovascular system has yet to be confirmed. The committee also noted that Lojuxta produces unwanted intestinal effects in most patients, sometimes of a magnitude that necessitates discontinuation of therapy, and that the medicine has resulted in an increase in enzyme levels in the liver of which they are not known the long-term consequences. Therefore, the committee established that these effects should be closely monitored and managed. Lojuxta was authorized in "exceptional circumstances" because complete information on Lojuxta could not be obtained due to the rarity of the disease. Every year the European Medicines Agency will review the new information available and this summary will be updated accordingly.
What information is still awaited for Lojuxta - lomitapide?
Since Lojuxta has been authorized in exceptional circumstances, the company that markets the medicine will carry out a long-term study in patients treated with Lojuxta in order to provide further data on its safety and efficacy, including its side effects on the liver, stomach, intestine and cardiovascular system. The study will also provide data on the pregnancies of women taking the medicine and on compliance by healthcare professionals with the recommendations to carry out screening and monitoring of patients before and during therapy.
What measures are being taken to ensure the safe and effective use of Lojuxta - lomitapide?
A risk management plan has been developed to ensure that Lojuxta is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Lojuxta, including the appropriate precautions to be followed by healthcare professionals and patients. In addition, the company that markets Lojuxta will provide all the doctors who could prescribe Lojuxta informational materials containing instructions on how to select suitable patients as well as basic safety information, including side effects, interactions with other medicines and use in women in fertile age. Information will also be provided to patients, including a brochure and an alert card.
Other information on Lojuxta - lomitapide
On 31 July 2013, the European Commission issued a marketing authorization for Lojuxta, valid throughout the European Union. For more information about treatment with Lojuxta, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist. Last update of this summary: 08-2013
