Real world data collection on the efficacy and drug usage of Raxone 150 mg 180 film-coated tablets for treatment of reduced vision in adolescent and adult patients with Leber’s hereditary optic neuropathy

Leber’s hereditary optic neuropathy (LHON) is a genetic disease that leads to sudden vision loss during young adult life. LHON is a disorder caused by mutations in the genetic code of the mitochondria, which are small subunits that reside within the cell. Mitochondria constantly convert energy locked in our food into energy that the cell can use. Brain cells, including the optic nerves, which are particularly energy-demanding, are therefore, more prone to damage if the mitochondria are not able to produce energy properly. A lack of energy production can lead to degeneration and death of retinal ganglion cells (RGCs), which are the nerve cells that communicate visual information to the brain. Loss of these cells leads to subsequent degeneration of the optic nerve and visual loss. An agreement with the applicant Chiesi for the temporary registration of a proprietary medicinal product on the list of reimbursable proprietary medicinal products, in accordance with the provisions of articles 112 to 117 inclusive of the royal decree of 1 February 2018 has been established with the working group for agreements (RIZIV/INAMI) and the Minister of Social Affairs. During the temporary reimbursement of 2 years, the company has to answer 6 questions (included in the agreement) to give an answer to the uncertainties that there are today.莱伯遗传性视神经病变（Leber’s hereditary optic neuropathy, LHON）是一种在青年时期引发突发性视力丧失的遗传性疾病。该病由线粒体内的基因突变所致，线粒体是存在于细胞内的微小细胞器。线粒体可持续将食物中储存的能量转化为细胞可利用的能量。 脑细胞（包括能量需求极高的视神经）若线粒体无法正常产能，则更易受损。能量产生不足会导致视网膜神经节细胞（retinal ganglion cells, RGCs）变性甚至死亡——这类神经细胞负责向大脑传递视觉信息。此类细胞的丧失会继发视神经变性与视力丧失。 我方已与协议工作组（RIZIV/INAMI）及社会事务大臣达成协议，针对申请人奇司（Chiesi）的一款专利药品，依据2018年2月1日皇家法令第112条至第117条（含首尾条款）的规定，将其纳入可报销专利药品名单并授予临时注册资格。在为期两年的临时报销期内，该企业需回应协议中列明的6项问题，以解答当前存在的各项不确定性。