8月8日，意大利生物制药公司Dompé重度神经营养性角膜炎（NK）治疗药物 Oxervate（cenegermin）赢得欧盟批准，神经营养性角膜炎是一种罕见的眼科疾病，其发病率不到万分之五。这次的批准对这家生物科技公司来说是一次重大胜利，因为这款药物2015年获得了孤儿药资格，迄今为止，该公司还没有一款生物技术药物获批用于这一适应证。
Dompé首席执行官Aringhieri称：神经营养性角膜炎是一种罕见疾病，直到现在患者没有合适的治疗选择。这次的批准对于受此疾病困扰的患者、该领域的研究及我们公司来说都是一个里程碑。欧洲监管机构的决定基于两项2期临床试验的阳性数据，试验受试者为 204 名罹患该疾病的患者。研究人员发现，与安慰剂相比，cenegermin用药患者经8周治疗后达到完全角膜愈合。
Aringhieri补充称：Oxervate是我们的第一个生物技术药物，它的获批归因于一支经验丰富并充满激情的团队的辛苦努力，他们从一开始就认准了这个项目。这是一个漫长、复杂而令人兴奋的旅程，整个旅程还没有结束，这只是这次重要监管步骤的一个开始。」这款产品将以 20 μg/mL 的滴眼液供患者使用，该公司的目标是让这款产品在欧洲以外的其它地区上市。
诺丁汉大学眼科教授兼诺丁汉大学医院顾问眼科医生Dua称：神经营养性角膜炎的病与三叉神经损伤有关，三叉神经损伤可导致角膜敏感性丧失，最严重情况下，它可引起溃疡、无菌性坏死和角膜穿孔，导致患者视力下降。鉴于这种疾病的严重性和可行性替代方案的缺乏，拥有一种可对角膜病变有效的方案对于眼科医师及患者本身是一个重大进展。
Oxervate (Cenegermin) - Augentropfen - DOMPE BIOTEC
Oxervate cenegermin
On 18 May 2017, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Oxervate, intended for the treatment of moderate to severe neurotrophic keratitis. Oxervate, which was designated as an orphan medicinal product on 14 December 2015, was reviewed under EMA’s accelerated assessment programme. The applicant for this medicinal product is Dompé farmaceutici S.p.A.
Oxervate will be available as a 20 microgram/ml eye drops solution. The active substance of Oxervate is cenegermin, a recombinant form of human nerve growth factor, which exerts a trophic effect and induces corneal epithelial cell growth and survival.
The benefits with Oxervate are its ability to stimulate corneal healing and restore ocular surface integrity in patients with neurotrophic keratitis suffering from persistent epithelial defects or corneal ulcers. The most common side effects are eye pain, eye inflammation, increased lacrimation and foreign body sensation in the eye.
The full indication is: "Treatment of moderate (persistent epithelial defect) or severe (corneal ulcer) neurotrophic keratitis in adults". It is proposed that Oxervate treatment be initiated and supervised by an ophthalmologist or a healthcare professional qualified in ophthalmology.
Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission. |
