Cadent Therapeutics, a company focused on the development of therapies to improve the lives of patients with movement and cognitive disorders, today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to CAD-1883, an investigational treatment for spinocerebellar ataxia , a genetic disorder characterized by progressive loss of coordination, slurred speech, difficulty controlling eye movements and cognitive dysfunction. Designed as a selective positive allosteric modulator of small-conductance, calcium-activated potassium ion channels , CAD-1883 has the potential to regulate neuronal firing and reduce disabilities in patients with SCA.
“We are pleased to obtain Orphan Drug Designation for CAD-1883 in SCA, a progressively debilitating disease for which there are currently no approved treatments,” said Jodie Morrison, Chief Executive Officer of Cadent Therapeutics. “This deeply underserved patient population deserves new therapies, and we look forward to advancing CAD-1883, our therapy that holds great potential for addressing their unmet needs.”
The FDA Office of Orphan Products Development grants Orphan Drug Designation to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. The designation allows Cadent to qualify for a number of incentives, including seven years of market exclusivity upon regulatory approval, if received; exemption from FDA application fees for spinocerebellar ataxia; and tax credits for qualified clinical trials.
Cadent Therapeutics ，一家致力于开发治疗方法以改善运动和认知障碍患者的生活的公司，今天宣布，美国食品药品监督管理局（Food and Drug Administration）已批准 Orapha 药物指定为 CAD-1883，一种研究治疗脊髓灰质炎的方法，一种遗传性疾病，其特征是逐渐丧失协调性、言语迟钝、难以控制眼球运动和认知功能障碍。CAD-1883作为选择性的小电导、钙激活钾离子通道的变构调制器，在 SCA 患者中具有调节神经元放电和减少残疾的潜力。
Cadent Therapeutics 首席执行官 Jodie Morrison 表示：“我们很高兴在 SCA 中获得 CAD-1883的孤儿药物指定，这是一种逐渐衰弱的疾病，目前尚无批准的治疗方法。”“这一严重得不到充分服务的患者群体应该得到新的治疗方法，我们期待着推进 CAD-1883，我们的治疗方法有很大的潜力来满足他们未得到满足的需求。”
FDA 孤儿产品开发办公室向药物和生物制剂授予孤儿药物名称，这些药物和生物制剂被定义为用于安全有效的治疗、诊断或预防影响美国少于20万人的罕见疾病/疾病的药物和生物制剂。包括七年的市场独占性经监管批准，如果收到；豁免从 FDA 申请费用的脊灰共济失调和税收抵免的合格临床试验。