intranasal carbetocin

CHICAGO, IL, January 18, 2022 (Newswire.com) – Levo Therapeutics, Inc., a biotechnology company dedicated to using genetic insights to advance treatments for Prader-Willi syndrome (PWS) and related disorders, announced today that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for LV-101 (intranasal carbetocin) as a treatment for hyperphagia, anxiousness, and distress associated with PWS.

FDA’s Division of Psychiatry concluded that, while LV-101 appears to be generally safe and well tolerated, the efficacy data available for the proposed 3.2 mg dose of LV-101 were insufficient for approval.  To address this issue, FDA recommended that an additional clinical study be conducted to confirm the results of the 3.2 mg dose.

Carbetocin is an analog of the naturally-occurring neuroendocrine hormone oxytocin.  Carbetocin was designed to have an improved receptor binding profile compared to oxytocin, with greater affinity for the oxytocin receptor and lower affinity for related vasopressin receptors.  Through our licensor, Ferring Pharmaceuticals, carbetocin is approved in over 90 countries outside the United States for the prevention of uterine atony and excessive bleeding during cesarean section delivery, as well as newly approved in the EU following vaginal birth, with an estimated cumulative exposure of over 10 million patients.  LV-101 is an investigational intranasal form of carbetocin, intended to be administered to patients with PWS three times each day before meals.  LV-101 has been granted orphan drug and Fast Track designations from the U.S. Food and Drug Administration (FDA).

Prader-Willi syndrome (PWS) is a complex, multisystem neurodevelopmental disorder that occurs in approximately 1 in 16,000 birthsi. The underlying cause of PWS is the lack of expression of paternally-inherited imprinted genes on chromosome 15q11-q13. These genetic anomalies lead to a distinctive phenotype that includes mild to moderate levels of intellectual disability, compulsivity, growth hormone deficiency, life-threatening hyperphagia, and anxiety.

"鼻饲卡贝缩宫素"

2022年1月18日（Newswire.com）--伊利诺伊州芝加哥市的Levo Therapeutics, Inc.生物技术公司致力于利用基因前景洞察力推进普拉德-威利综合征（PWS）及相关疾病治疗。它收到美国食品和药物管理局（FDA）关于其"鼻饲卡贝缩宫素"作为治疗PWS相关的过度吞咽、焦虑和痛苦的新药申请（NDA）的回应函。 FDA的精神病部门认为，尽管"鼻饲卡贝缩宫素"似乎普遍安全且耐受性良好，但3.2毫克剂量的"鼻饲卡贝缩宫素"的疗效数据不足以批准。FDA建议进行一项额外的临床研究以确认3.2毫克剂量。

卡贝缩宫素是一种自然存在的神经内分泌激素催产素的类似物。 与催产素相比，卡贝缩宫素被设计成具有更好的受体结合特性，对催产素受体的亲和力更大，而对相关的加压素受体的亲和力更低。 "鼻饲卡贝缩宫素"是一种研究性的卡贝缩宫素的鼻内制剂，每天给PWS患者在饭前用三次。"鼻饲卡贝缩宫素"已被美国食品和药物管理局（FDA）授予孤儿药和快速通道的称号。

普拉德-维利综合征（PWS）是一种复杂的、多系统的神经发育障碍，大约每16,000个新生儿中就有一个发生。PWS的根本原因是15q11-q13号染色体上父亲遗传的印记基因缺乏表达。这些基因异常导致了一种独特的表型，包括轻度到中度的智力障碍、强迫症、生长激素缺乏、危及生命的多食症和焦虑症。