继7月底获得美国FDA批准之后，瑞士制药巨头诺华（Novartis）开发的抗癌药Odomzo（sonidegib，200mg）近日再获欧盟批准，用于不适合手术治疗或放射治疗的局部晚期基底细胞癌（laBCC）成人患者的治疗。此前，Odomzo已获得澳大利亚和瑞士批准，并于今年6月25日获得欧盟CHMP建议批准的积极意见。
基底细胞癌（BCC）是一种最常见的皮肤癌，约占非黑色素瘤皮肤癌的80%以上，该病常发于头颈部，鼻子是最常见部位。局部晚期是指BCC从最初部位扩散至附近组织，可导致高度毁容，从身体和精神双方面对患者带来严重影响。晚期BCC约占BCC病例的1-10%。尽管BCC通常可在早期阶段确诊并治疗，但约3%的患者在治疗5年后病情复发。由于人口老龄化、紫外线暴露增加等因素，BCC的发病率每年上升10%左右，该病在全球范围内的发病率预计在0.003%-0.55%。
尽管BCC很少发展为晚期，但一旦进展到这一阶段几乎没有治疗选择。Odomzo将为局部晚期BCC群体提供一种新的非侵入性治疗方案，为这类患者带来新的生活希望。
Odomzo的获批，是基于一项国际多中心随机双盲II期BOLT研究中所取得的可持续的客观缓解率（ORR）数据。该研究在不适合局部治疗的局部晚期基底细胞癌（laBCC）或转移性基底细胞癌（mBCC）成人患者中开展，评估了Odomzo 200mg和800mg剂量的疗效和安全性。经中心审查委员会评定的数据显示，200mg剂量组客观缓解率（ORR）为56%，其中完全缓解率（CR）为5%，部分缓解率（PR）为52%；经研究人员评定的数据显示ORR为71%，其中CR占9%，PR占62%。目前，中位缓解持续时间尚未达到。中心审查委员会评定的中位无进展生存期为22个月，研究人员评定的无进展生存期为19个月。
此外，无证据表明800mg剂量组ORR数据好于200mg剂量组。
Odomzo（sonidegib，原名LDE225）是一种口服选择性Smoothened（SMO）抑制剂。SMO是一种7次跨膜蛋白，调控Hedgehog（Hh）信号通路，该通路在干细胞维持、组织修复、晚期基底细胞癌中发挥关键作用。目前，诺华正在调查sonidegib用于多种疾病的治疗，包括骨髓纤维化、白血病及实体瘤（如胰腺癌、乳腺癌和非小细胞肺癌）。

Novartis drug Odomzo gains EU approval for locally advanced basal cell carcinoma, providing new non-invasive therapy for patients
Thursday, 20 August 2015 - 8:15am
Approval follows positive CHMP opinion based on pivotal Phase II study showing durable objective response rate per central review of 56% in patients with laBCC
 Basal cell carcinoma is the most common form of skin cancer and can be highly disfiguring and invasive at advanced stages
 Odomzo (sonidegib), a smoothened inhibitor, is already approved in the US, Australia and Switzerland, with additional regulatory filings underway worldwide
Basel, August 20, 2015 - Novartis announced today that the European Commission has approved Odomzo (sonidegib, formerly LDE225) 200 mg capsules for the treatment of adult patients with locally advanced basal cell carcinoma (laBCC) who are not amenable to curative surgery or radiation therapy.

"I have seen first-hand the devastating impact advanced basal cell carcinoma can have on those living with the disease. As the lesions are usually highly visible and located predominantly on the face, they can impact patients both physically and emotionally," said Reinhard Dummer, MD, Professor and Vice Chairman, Department of Dermatology at the University of Zurich. "The approval of Odomzo brings new hope in the form of a non-invasive option to help treat this disfiguring and potentially life-threatening disease."

Basal cell carcinoma (BCC) consists of abnormal, uncontrolled growths or lesions that arise in the skin's basal cells, which line the deepest layer of the epidermis (the outermost layer of the skin) and accounts for more than 80% of non-melanoma skin cancers. Advanced BCC is thought to represent roughly 1-10% of all cases of BCC-. Although BCC rarely becomes advanced, there have been few treatment options at this stage of the disease. Worldwide incidence of BCC is rising by 10% each year due to factors such as an aging population and increased ultraviolet exposure.