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Aegerion高胆固醇血症新药Lojuxta获EMA批准

来源:生物谷 2013-08-05 21:27

2013年8月5日讯 /生物谷BIOON/ --美国生物制药公司Aegerion制药今天宣布,欧洲药品管理局(EMA)已批准将Lojuxta(lomitapide)硬胶囊作为低脂肪饮食及其他降脂药用产品的一种辅助药物,用于纯合子家族性高胆固醇血症(HoFH)成人患者的治疗。

纯合子家族性高胆固醇血症(HoFH)是一种极罕见的常染色体显性遗传性疾病,发病机制为细胞膜表面的低密度脂蛋白(LDL)受体缺如或异常,导致体内LDL代谢异常,造成血浆总胆固醇(TC)水平和低密度脂蛋白-胆固醇(LDL-C)水平升高,往往导致极其严重的心血管问题。(生物谷Bioon.com)

英文原文:Aegerion gains European approval for rare disease drug

The European Commission has approved US biopharma company Aegerion Pharmaceuticals’ (Nasdaq: AEGR) Lojuxta (lomitapide) hard capsules as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein (LDL) apheresis in adult patients with homozygous familial hypercholesterolemia (HoFH), a rare and serious medical condition.

"The approval of Lojuxta marks an important day for patients in Europe with HoFH, and is a major step for Aegerion in our goal to bring therapy to patients globally," said Marc Beer, chief executive of Aegerion, adding: "We are now keenly focused on launch, and are actively working with national health authorities in an effort to make Lojuxta available as swiftly as possible."

Alberico Catapano, president of the European Atherosclerosis Society quoted by Aegerion, commented: "Despite the treatments we have available today, patients continue to experience severe elevations in LDL-C. With novel therapies such as Lojuxta it is possible to achieve target levels in LDL-C for patients with HoFH. This marks a change in our ability in daily practice to truly impact the lives of these patients."

The European Commission's decision is based on a favorable benefit-risk assessment from Aegerion's Phase III study which evaluated the safety and efficacy of the medicine to reduce LDL-C levels in 29 adult patients with HoFH,, and follows a unanimous positive opinion earlier this year from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP; The Pharma Letter May 31).

Risk management plan adopted

Aegerion has adopted a risk management plan to help educate physicians on the safety information for Lojuxta and appropriate precautions to be followed by health care professionals and patients. In addition, as part of Aegerion's post-marketing commitment to both the US Food and Drug Administration and the EMA, the company will conduct an observational cohort study to generate more data on the long-term safety profile of lomitapide, the patterns of use and compliance and the long-term effectiveness of controlling LDL-C levels.

Under the trade name Juxtapid, the drug was approved by the FDA late last year (TPL December 28, 2012). Lomitapide may generate at least $300 million in annual sales, said William Tanner, an analyst at Lazard Capital Markets said at the time of the US approval. The estimate is based on a price of $300,000 a year per patient that is similar to costs of other drugs for diseases with few treatment options, he said. Kimberly Lee, a Think Equity Partners analyst, said peak sales may reach $450 million.

Will compete with Kynamro

Aegerion recently reported that sales of Juxtapid, its only marketed drug, totaled $6.5 million in the second quarter of this year, well above Wall Street estimates. For the full year Aegerion is forecasting between $30 million and $35 million in revenue, up from previous estimates of $15-$25 million. Juxtapid competes with French drug major Sanofi (Euronext: SAN0 and Isis Pharmaceuticals’ (Nasdaq: ISIS) Kynamro (mipomersen sodium), another drug for HoFH, which was approved in the USA early this year (TPL January 30).

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