近日，美国FDA批准胰腺酶新药Ultresa(pancrelipase)为缓释胶囊，适用于成人和儿童，主要用于治疗因患有囊性纤。
Ultresa（胰脂肪酶 pancrelipase）延迟释放胶囊是由胰脂肪酶，来自猪胰腺的提取物组成的胰酶制剂。胰脂肪酶含有多种酶类，包括猪来源的脂肪酶，淀粉酶和蛋白酶。Ultresa中的胰腺酶类似于由胰腺生理分泌的消化酶。
Ultresa专门用于治疗由于囊性纤维化或其他病症引起的外分泌胰腺功能不全。
Ultresa以口服给药的胶囊形式提供。Ultresa的初始剂量应该从最低推荐剂量开始并逐渐增加。Ultresa的剂量应基于临床症状，存在的脂肪程度和饮食的脂肪含量而个体化。
批准日期：2012年3月1日 公司：Aptalis Pharma US, Inc
ULTRESA（胰脂肪酶 pancrelipase）延迟释放胶囊，用于口服使用
美国初步批准：2012年
作用机制
在ULTRESA中的胰酶催化脂肪水解为单酸甘油酯，甘油和游离脂肪酸，蛋白质成肽和氨基酸，并且在十二指肠和近端小肠中淀粉成糊精和短链糖，例如麦芽糖和麦芽三糖，由此起作用 由胰腺生理分泌的消化酶。
适应症和用法
ULTRESA是猪来源的脂肪酶，蛋白酶和淀粉酶的组合，指示用于治疗由于囊性纤维化或其他病症引起的外分泌胰腺功能不全。
剂量和给药
ULTRESA不与任何其他胰脂肪酶产品互换。
用法
ULTRESA应该整个吞下。不要粉碎或咀嚼胶囊和胶囊内容物。对于不能吞咽完整胶囊的儿童或患者，可将内容物喷洒在pH为4.5或更低的软酸性食物上，例如苹果酱或酸奶。
婴儿（长达12个月）
婴儿可以每120mL配方或每次母乳喂养给予4,000个脂肪酶单位。
在给药前不要将ULTRESA胶囊内容物直接混入配方奶粉或母乳中。
儿童年龄大于12个月和小于4岁
以每个膳食1,000个脂肪酶单位/kg体重开始，每个膳食最多2,500个脂肪酶单位/kg体重（或小于或等于10,000个脂肪酶单位/kg体重每天）或小于4,000个脂肪酶单位/g脂肪每天摄入。
儿童4岁及以上和成人
从每个膳食500个脂肪酶单位/kg体重开始，到每个膳食最多2,500个脂肪酶单位/ kg体重（或小于或等于10,000个脂肪酶单位/kg体重每天），或小于4,000个脂肪酶单位/g脂肪每天摄入。
剂量限制
剂量不应超过囊性纤维化基金会共识会议指南中规定的建议最大剂量。
剂量形式和强度
延迟释放胶囊：4,000 USP单位的脂肪酶; 8,000 USP单位的蛋白酶; 8,000 USP单位的淀粉酶
延迟释放胶囊：13,800USP单位的脂肪酶; 27,600 USP单位的蛋白酶; 27,600 USP单位的淀粉酶
延迟释放胶囊：20,700 USP单位的脂肪酶; 41,400USP单位的蛋白酶; 4100USP单位的淀粉酶
延迟释放胶囊：23,000 USP单位的脂肪酶; 46,000 USP单位的蛋白酶; 46,000 USP单位的淀粉酶
禁忌症
没有。
警告和注意事项
纤维结肠病与胰腺酶替代的高剂量使用有关。当ULTRESA的剂量超过每千克体重2,500脂肪酶单位/每餐（或大于10,000脂肪酶单位/ kg体重每天）时要小心。
为了避免刺激口腔粘膜，不要嚼ULTRESA或留在口中。
在对患有痛风，肾损伤或高尿酸血症的患者开具ULTRESA时应小心。
存在所有胰腺酶产物（包括ULTRESA）的病毒传播的理论风险。
向对猪来源的蛋白质具有已知过敏反应的患者施用胰脂肪酶时要小心。
不良反应
最常见的不良反应（≥ULTRESA治疗的患者的≥7％）是头痛，咽喉疼痛和鼻出血。

U.S. Commercial Availability of ULTRESA(Pancrelipase) Delayed-Release Capsules
U.S., Inc., a global specialty pharmaceutical company focused on gastrointestinal diseases and cystic fibrosis (CF), announced that ULTRESA™ (pancrelipase) delayed-release capsules are now commercially available in the U.S. ULTRESA is a combination of porcine-derived lipases, proteases, and amylases indicated for the treatment of exocrine pancreatic insufficiency (EPI) due to CF or other conditions.
Frank Verwiel, M.D., President and Chief Executive Officer of Aptalis Pharma, stated, "Aptalis is pleased to be able to make ULTRESA available in the U.S. With ULTRESA, patients and their healthcare providers will have another important treatment option for EPI due to CF or other conditions. ULTRESA gives us a new opportunity to support the CF and gastrointestinal disorder communities and represents another important step toward the mission that inspires us: to improve health and quality of care by providing specialty therapies for patients around the world."
ULTRESA helps treat malabsorption in patients with EPI due to CF or other conditions by significantly improving protein and fat absorption versus placebo.
In the pivotal, placebo-controlled study of 31 patients with EPI due to CF, ULTRESA-treated patients had a significantly higher coefficient of fat absorption (CFA, primary endpoint) and protein absorption (secondary endpoint) than placebo. Mean CFA was 89% with ULTRESA treatment compared to 56% with placebo treatment. The mean difference in CFA was 35 percentage points in favor of ULTRESA treatment (95% CI: (25, 45); p < .0001). The mean coefficient of nitrogen absorption (CNA) was 84% with ULTRESA compared to 59% with placebo treatment. The mean difference in CNA was 26 percentage points in favor of ULTRESA (95% CI: (18,33); p < .0001).
ULTRESA is available in three capsule strengths (23,000, 20,700, and 13,800 USP units of lipase) containing enteric-coated mini tablets. The 23,000 unit high dosage strength capsule may allow patients to take fewer capsules to achieve the prescribed dose per meal.
The ULTRESA Patient Support Program will also be available to eligible patients at launch.
ULTRESA is well tolerated. The most common adverse reactions occurring in at least two patients treated with ULTRESA were headache, pharyngolaryngeal pain, and epistaxis.
Important Safety Information for ULTRESA™
•Fibrosing colonopathy is associated with high-dose use of pancreatic enzyme replacement. Exercise caution when doses of ULTRESA exceed 2,500 lipase units/kg of body weight per meal (or greater than 10,000 lipase units/kg of body weight per day).
•To avoid irritation of oral mucosa, do not chew ULTRESA or retain in mouth.
•Exercise caution when prescribing ULTRESA to patients with gout, renal impairment, or hyperuricemia.
•There is theoretical risk of viral transmission with all pancreatic enzyme products, including ULTRESA.
•In rare cases, patients taking pancreatic enzyme products with different formulations of the same active ingredient (pancrelipase) have experienced severe allergic reactions including anaphylaxis, asthma, hives, and pruritus.
•Exercise caution when administering pancrelipase to a patient with a known allergy to proteins of porcine origin.
•The most common adverse reactions(≥ 7% of patients treated with ULTRESA) were headache, pharyngolaryngeal pain, and epistaxis.
•Use of ULTRESA in pediatric patients is limited by the available capsule dosage strengths and their ability to provide the recommended dose based on age and weight.
•ULTRESA is not interchangeable with any other pancrelipase product.
For complete information about safety, warnings and precautions for ULTRESA™, please click here for the full Prescribing Information and Medication Guide.
A multicenter, randomized, double-blind, placebo-controlled crossover study of 31 patients, aged 8 to 37, with EPI due to CF or other conditions. The final analysis population was comprised of 24 patients who completed both treatment periods and had stool results available for each treatment period. Patients were randomized to receive ULTRESA (at a dose not to exceed 2500 lipase units per kilogram per meal or snack) or matching placebo for six to seven days of treatment followed by crossover to the alternate treatment for an additional six to seven days. All patients consumed a high-fat diet during the treatment periods. The CFA and CNA were determined by a 72-hour stool collection during both treatment periods.
About Aptalis
Aptalis Pharma is a privately held, leading specialty pharmaceutical company providing innovative, effective therapies for unmet medical needs including cystic fibrosis and gastrointestinal disorders. Aptalis has manufacturing and commercial operations in the United States, the European Union and Canada, and its products include ZENPEP®, CANASA®, CARAFATE®, PYLERA®, RECTIV®, VIOKACE™, ULTRESA™ LACTEOL®, DELURSAN®, PANZYTRAT®, and SALOFALK®. Aptalis also formulates and clinically develops enhanced pharmaceutical and biopharmaceutical products for itself and others using its proprietary technology platforms including bioavailability enhancement of poorly soluble drugs, custom release profiles, and taste-masking/orally disintegrating tablet (ODT) formulations. For more information, visit.
https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=73861076-218d-4f8e-872d-0e0690c374a8