FDA Approves Scenesse


2019-10-09 18:47:12 Drugs


All News Consumer Pro New Drugs Pipeline Clinical Trials FDA Alerts More FDA Approves Scenesse (afamelanotide) for Erythropoietic Protoporphyria October 08, 2019 -- The U.S. Food and Drug Administration today granted approval to Scenesse (afamelanotide) to increase pain-free light exposure in adult patients with a history of phototoxic reactions (damage to skin) from erythropoietic protoporphyria. For patients who are suffering from erythropoietic protoporphyria, a rare disorder, exposure to light may be extremely painful. Prior to today’s approval, there were no FDA-approved treatments to help erythropoietic protoporphyria patients increase their light exposure,” said Julie Beitz, M.D., director of FDA’s Center for Drug Evaluation and Research Office of Drug Evaluation III. “Today’s approval is one example of the FDA’s ongoing commitment to encourage industry innovation of therapies to treat rare diseases, and work with drug developers to make promising new therapies available to patients as safely and efficiently as possible.” Erythropoietic protoporphyria is a rare disorder caused by mutations leading to impaired activity of ferrochelatase, an enzyme involved in heme production. Heme is an important component in hemoglobin, the oxygen carrying molecule in red blood cells. The decrease in ferrochelatase activity leads to an accumulation of protoporphyrin IX (PPIX) in the body. Light reaching the skin can react with PPIX causing intense skin pain and skin changes, such as redness and thickening. Scenesse (afamelanotide), a melanocortin-1 receptor (MC1-R) agonist, increases the production of eumelanin in the skin independent of exposure to sunlight or artificial light sources.  It is an implant that is administered subcutaneously (inserted under the skin).  The efficacy of Scenesse was established in two parallel group clinical trials with patients with erythropoietic protoporphyria who received Scenesse or placebo form of the implant subcutaneously every two months. The first clinical trial enrolled 93 subjects, of whom 48 received Scenesse, and were followed for 180 days. The primary endpoint was the total number of hours over 180 days spent in direct sunlight between 10 a.m. and 6 p.m. on days with no pain. The median total number of hours over 180 days spent in direct sunlight between 10 a.m. and 6 p.m. on days with no pain was 64 hours for patients receiving Scenesse and 41 hours for patients taking placebo. The second clinical trial enrolled 74 patients, of whom 38 received Scenesse, and were followed for 270 days. The primary endpoint was the total number of hours over 270 days spent outdoors between 10 am and 3 pm on days with no pain for which “most of the day” was spent in direct sunlight. The analysis did not include sun exposure on days patients reported spending time in a combination of both direct sunlight and shade. The median total number of hours over 270 days spent outdoors between 10 am and 3 pm on days with no pain for which “most of the day” was spent in direct sunlight was six hours for patients receiving Scenesse and 0.75 hours for patients receiving placebo.  Scenesse’s most common side effects are implant site reaction, nausea, oropharyngeal (part of the throat just behind the mouth, where the oral cavity starts) pain, cough, fatigue, skin hyperpigmentation, dizziness, melanocytic nevus (moles), respiratory tract infection, somnolence (feeling drowsy), non-acute porphyria (build-up of normally occurring molecules created during heme production) and skin irritation. Scenesse should be administered by a health care professional who is proficient in the subcutaneous implantation procedure and has completed the applicant-provided training. Scenesse may induce skin darkening, and a full body skin examination is recommended for patients twice a year. In addition, patients are encouraged to maintain sun protection measures during treatment with Scenesse to prevent phototoxic reactions related to erythropoietic protoporphyria. The FDA granted this application Priority Review designation. Scenesse also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.The approval of Scenesse was granted to Clinuvel. Source: FDA Posted: October 2019 Related Articles: US FDA Review for Scenesse Extended 3 Months - June 3, 2019 Clinuvel Completes Scenesse FDA Filing - June 25, 2018 Historic New Drug Application for the use of Scenesse (afamelanotide) in Rare Metabolic Disorder Erythropoietic Protoporphyria (EPP) - June 25, 2018 Scenesse (afamelanotide) FDA Approval History
FDA 批准用于促红细胞生成性原卟啉的场景 2019年10月8日——美国食品药品监督管理局(Food and Drug Administration)今天批准 Scenes (阿非美拉那肽)增加成年患者的无痛光暴露,这些患者有红血球原卟啉引起的光毒性反应(皮肤损伤)。 对于患有红血球原卟啉症(一种罕见的疾病)的患者,暴露在阳光下可能是非常痛苦的。在今天的批准之前,没有 FDA 批准的治疗方法来帮助促红细胞生成性原卟啉症患者增加他们的光暴露,” Julie Beitz 医学博士(M.D.)博士说,他是 FDA 药物评估和研究中心的主任。“今天的批准是 FDA 持续致力于鼓励治疗罕见疾病的疗法行业创新的一个例子,并且与药物开发商合作,尽可能安全、高效地为患者提供有希望的新疗法。” 促红细胞生成素原卟啉是一种罕见的疾病引起的突变,导致损害的活动铁蛋白酶,一种酶参与了血红素的生产。血红蛋白是血红蛋白的重要组成部分,血红蛋白是红细胞中携带氧的分子.铁化酶活性的降低导致体内原卟啉 IX ( PPIX )的积累。光到达皮肤可以与 PPIX 反应,造成强烈的皮肤疼痛和皮肤变化,如红肿和增厚。场景( afamelanotide )是一种黑素皮质激素-1受体( MC1-R )激动剂,它在不受阳光或人工光源照射的情况下增加了皮肤中的真黑色素的产生。它是皮下注射的植入物(插入皮肤下)。 在两组平行临床试验中建立了 Sceness 的疗效,每两个月接受一次假体或安慰剂形式的红血球原卟啉患者。首次临床试验共纳入93例受试者,其中48例接受了 Scenest 治疗,随访180天。主要终点为每天上午10时至下午6时期间在直接阳光下度过的180天以上的总小时数。在没有疼痛的情况下,每天上午10时至下午6时在阳光直射中度过180天以上的平均总小时数为接受情景治疗的患者64小时,服用安慰剂的患者41小时。 第二次临床试验共纳入74例患者,其中38例接受了 Scenest 治疗,随访270天。主要终点是每天上午10时至下午3时在室外花费270天以上的总小时数,没有任何疼痛,“大部分时间”是在直接阳光下度过的。该分析没有包括白天暴露在阳光下的病人报告的花费时间在直接阳光和阴影的结合。每天上午10时至下午3时在户外度过的270天中,没有疼痛的“大部分时间”是在阳光直射下度过的,其中接受情景治疗的患者平均6小时,接受安慰剂的患者平均0.75小时。 场景最常见的副作用是植入部位反应、恶心、口咽(口腔后部的喉部,口腔开始处)疼痛、咳嗽、疲劳、皮肤色素沉着、头晕、黑色素细胞痣(痣)、呼吸道感染、嗜睡(感觉昏昏昏欲睡)。非急性卟啉(血液生成过程中产生的正常发生的分子的积累)和皮肤刺激。情景应由精通皮下植入程序并已完成申请人提供的培训的卫生保健专业人员进行管理。场景可能会导致皮肤变黑,建议每年对患者进行两次全身皮肤检查。此外,鼓励患者在治疗过程中保持防晒措施,以防止与红血球原卟啉有关的光毒性反应。 FDA 授予了这项申请优先审查的称号。场景还获得了孤儿药物的指定,这提供了激励措施,以协助和鼓励发展药物的罕见疾病。场景的批准授予了 Clinuvel 。