2014年9月25日訊 /生物谷BIOON/ --生物技術巨頭新基（Celgene）在2013年憑藉其明星藥物Abraxane（注射用紫杉醇[白蛋白結合型]）著實風光了一把，該藥是一種化療藥物，因賈伯斯之死和癌中之王——胰腺癌適應症而名聲大噪。今年，Celgene又收穫了一枚重磅藥物Otezla，該藥6個月內接連收穫2個適應症，與市售藥物相比，Otezla具有無需監測及口服的巨大優勢，業界預測，Otezla的年銷售峰值將突破20億美元。
新基（Celgene）近日宣布，口服藥物Otezla（apremilast）獲FDA批准，用於適合光療和系統療法的中度至重度斑塊型銀屑病（Plaque Psoriasis）成人患者的治療。Otezla是一種口服、選擇性磷酸二酯酶4（PDE4）抑制劑，該藥是FDA批准的首個也是唯一一個用於斑塊型銀屑病治療的PDE4抑制劑。
Otezla將為廣泛的斑塊型銀屑病患者群體提供一種有價值的治療選擇，包括以前使用過生物製劑或常規系統性藥物治療的患者群體。銀屑病（psoriasis）是一種由不受控免疫反應導致的皮膚慢性炎症性疾病，全球患者總數超過1.25億人。
Otezla的獲批，是基於2項研究（ESTEEM-1和ESTEEM-2）的主要療效和安全性數據。這2項研究均為多中心、隨機、雙盲、安慰劑對照研究，在中度至重度斑塊型銀屑病（Plaque Psoriasis）成人患者中開展。研究中，Otezla使患者斑塊型銀屑病病情取得了顯著且具有臨床意義的改善。此前，FDA已於今年3月批准Otezla用於活動性銀屑病性關節炎（PsA）成人患者的治療。
Otezla銷售峰值超20億美元
業界認為，儘管面臨著注射型藥物腫瘤壞死因子（TNF）抑制劑的競爭，尤其是艾伯維的Humira（阿達木單抗）和輝瑞/安進的Entrel（etanercept），但Otezla用藥不需要常規的實驗室監測，且是一種口服藥物，相比市售藥物，Otezla具有巨大優勢，將為患者和醫生提供一種重要的治療選擇。
EvaluatePharma此前預測，Otezla在2018年的銷售額將達到12.19億美元，而此次FDA批准Otezla新適應症，業界預期，Otezla的銷售峰值有望突破20億美元。
關於Otezla（apremilast）：
Otezla（apremilast）是一種口服小分子磷酸二酯酶(PDE4)抑制劑，在細胞內調控促炎症和抗炎介質的網絡。PDE4是一種環磷酸腺苷(cAMP)特異性PDE，是炎性細胞中主要的PDE。PDE4抑制可提升細胞內cAMP水平，通過調控TNF-α、IL-23和其他炎性細胞因子的表達相應下調炎性反應。cAMP升高也會增加抗炎細胞因子，例如IL-10。（生物谷Bioon.com）
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英文原文：Oral OTEZLA? (apremilast) Approved by the U.S. Food and Drug Administration for the Treatment of Patients with Moderate to Severe Plaque Psoriasis
In phase III studies, OTEZLA resulted in significant and clinically meaningful improvements in plaque psoriasis
OTEZLA demonstrated a consistent safety and tolerability profile across clinical trials
SUMMIT, N.J.--(BUSINESS WIRE)-- Celgene Corporation (NASDAQ: CELG) today announced that the U.S. Food and Drug Administration (FDA) has approved OTEZLA? (apremilast), the Company's oral, selective inhibitor of phosphodiesterase 4 (PDE4), for the treatment of patients with moderate to severe plaque psoriasis for whom phototherapy or systemic therapy is appropriate. OTEZLA is the first and only PDE4 inhibitor approved for the treatment of plaque psoriasis. Psoriasis, a chronic inflammatory disease of the skin resulting from an uncontrolled immune response, affects more than 125 million people worldwide.
"OTEZLA offers an important new treatment option for patients whose symptoms are not adequately improving with their current treatments. In clinical trials, OTEZLA reduced redness, thickness, and scaliness of plaques in patients with moderate or severe plaque psoriasis," said Dr. M. Shane Chapman, Section Chief of Dermatology at Dartmouth-Hitchcock Medical Center. "Because the product labeling does not require routine laboratory monitoring, oral OTEZLA may be a welcome new option for patients and physicians looking for a different treatment experience."
The approval of OTEZLA was based primarily on safety and efficacy results from two multi-center, randomized, double-blind, placebo-controlled studies - ESTEEM 1 and ESTEEM 2 - conducted in adult patients with moderate to severe plaque psoriasis: body surface area (BSA) involvement of ≥10%, static Physician Global Assessment (sPGA) of ≥3 (moderate or severe disease), Psoriasis Area and Severity Index (PASI) score ≥12, and candidates for phototherapy or systemic therapy.
"OTEZLA offers a valuable treatment option for a spectrum of plaque psoriasis patients - patients who are treatment-na?ve as well as patients who are treatment-experienced, including those previously treated with biologic agents or conventional systemic agents," said Scott Smith, President Inflammation & Immunology for Celgene Corporation. "The FDA approval of OTEZLA for plaque psoriasis, together with the previous approval for psoriatic arthritis, reflects Celgene's commitment to extending the reach of our research and science in an effort to improve the lives of people worldwide living with chronic inflammatory diseases."
In the ESTEEM studies, OTEZLA treatment resulted in significant and clinically meaningful improvements in plaque psoriasis as measured by PASI scores at week 16. Clinical improvement as measured by sPGA scores of clear to almost clear were also demonstrated in both studies.
The safety of OTEZLA was assessed in 1,426 patients from three clinical trials. Side effects of OTEZLA were diarrhea, nausea, upper respiratory tract infection, tension headache, and headache. Before starting OTEZLA, patients should inform their doctor if they have a history of depression or suicidal behavior and if these conditions or other mood changes develop or worsen while taking OTEZLA. Patients taking OTEZLA should have their weight checked regularly.
"Psoriasis is a serious autoimmune disorder commonly associated with comorbidities," said Randy Beranek, president and CEO, National Psoriasis Foundation. "Effectively treating psoriasis is an important part of managing a patient's overall health. Having a new treatment like OTEZLA is important so patients can have more options and can work closely with their providers to find what works best for them."
OTEZLA? is available in the U.S. and is dispensed through a comprehensive network of specialty pharmacies. For more information about OTEZLA distribution and the exclusive treatment support services (including reimbursement assistance and 24/7 nurse support), doctors and patients can contact Otezla SupportPlus? at 1-844-4OTEZLA (1-844-468-3952) or visit www.OTEZLA.com for more information.
OTEZLA was approved on March 21, 2014 by the U.S. Food and Drug Administration (FDA) for the treatment of adults with active psoriatic arthritis. A New Drug Submission (NDS) for psoriatic arthritis was submitted to health authorities in Canada in the second quarter of 2013. A NDS for psoriasis in Canada as well as a combined psoriatic arthritis/psoriasis Marketing Authorization Application (MAA) in Europe were all submitted to health authorities in the fourth quarter of 2013.