2015年5月28日，美国FDA批准Rapamune(Sirolimus，西罗莫司)治疗淋巴管平滑肌增生症(LAM)，这是一种罕见的侵袭性肺部疾病，主要影响生育年龄的妇女。这是获批用于治疗该疾病的首款药物。
LAM的特点是侵犯肺组织(包括呼吸道)及血管/淋巴管的平滑肌细胞异常增生，可引起肺损害，导致呼吸道阻塞及限制氧气输送到人体。LAM是一种非常罕见的疾病。
据美国国家医学图书馆提供的信息称，全球每百万妇女中只有2%到5%的人已知患有这种疾病。
Rapamune有片剂与口服溶液两种剂型供人们使用，这款药物最初在1999年作为一种免疫抑制剂获批用于帮助13岁及以上年龄接受肾移植的患者阻止器官排斥反应。由于Rapamune的申请者证明这款药物与现有治疗药物相比可提供实质性的改善，这款药物从而获得突破性治疗药物资格。
它还获得了优先审评权，优先审评权可为那些在一种严重疾病或病症治疗中可能对安全性或有效性有明显改善的药物提供一个加快的审评。由于LAM是一种罕见疾病或病症，Rapamune用于这一适应症也获得了孤儿药资格。这款药物的开发在某种程度上也获得了FDA孤儿产品捐助计划的支持，该计划可以为用于罕见疾病或症状产品的安全性和/或有效性临床研究提供援助。
不同的FDA计划，如孤儿产品资格及突破性治疗药物资格，它们为申请者提供了财政激励，增加了互动并获得来自FDA的建议，促进了用于罕见病创新治疗药物的开发与批准，否则是不可能达到的，」FDA药物评价与研究中心新药办公室主任、医学博士Jenkins称。这种特别计划使FDA帮助药品生产商将拯救生命的药物以尽快的速度送到需要的患者手中成为可能。
Rapamune治疗LAM的安全性与有效性在一项Rapamune 与一种非活性药物（安慰剂）进行对照的临床试验中得到研究，试验中共有89名受试者，他们先进行12个月的治疗，随后是12个月的观察期。主要终点是一移钟内用力呼吸（一秒钟用力呼气容积或 FEV1）期间人所能呼出气体变化率的组间差异。
在12个月的治疗期间内，FEV1的差异平均下降大约153mL。在停止使用Rapamune后，肺功能以类似于安慰剂组的速率继续下降。
最常报道的与Rapamune治疗LAM相关的副作用是嘴与嘴唇溃疡、腹泻、腹痛、恶心、咽喉痛、痤疮、胸痛、腿肿胀、上呼吸道感染、头痛、眩晕、肌肉疼痛和高胆固醇。严重副作用包括过敏症及在肾移植患者中观察到肿胀（水肿）。Rapamune由宾夕法尼亚州费城的Wyeth制药制造，该公司是辉瑞旗下的一个子公司。

Indications and Usage
RAPAMUNE is indicated for the prevention of organ rejection in kidney transplant patients aged 13 years or older. Blood levels of sirolimus should be checked in all patients taking Rapamune.
In patients at low to moderate risk of acute rejection, it is recommended that Rapamune be used initially in combination with cyclosporine and corticosteroids; cyclosporine should be withdrawn approximately 3 months after transplantation. Cyclosporine withdrawal has not been studied in patients who have had severe acute rejection prior to cyclosporine withdrawal, those who require dialysis or have a high serum creatinine, Black patients, patients receiving a repeat kidney transplant, patients receiving other transplanted organs besides the kidney transplant, or patients with antibodies that may be directed against the kidney transplant.
In patients at high risk of acute rejection (defined as Black patients and/or patients receiving a repeat kidney transplant who lost a previous kidney transplant from rejection and/or patients with high levels of antibodies that may be directed against the kidney transplant), it is recommended that Rapamune be used in combination with cyclosporine and corticosteroids for the first year following transplantation. The safety and efficacy of this combination in high-risk patients have not been studied beyond one year; therefore, after the first year, adjustments to the immunosuppressive regimen may be considered by your doctor.
In pediatric patients, the safety and efficacy of Rapamune have not been established in kidney transplant patients less than 13 years old, or in patients less than 18 years old who are considered at high risk of acute rejection.
The safety and efficacy of Rapamune without cyclosporine in newly transplanted kidney patients have not been established.
The safety and efficacy of changing from either cyclosporine or tacrolimus to Rapamune in maintenance kidney transplant patients have not been established.
Important Safety Information
There is an increased risk of developing infections or certain cancers, especially lymphoma and skin cancers. Rapamune has not been shown to be safe and effective in people who have had liver or lung transplants. Serious complications and death may happen in people who take Rapamune after a liver or lung transplant. You should not take Rapamune if you have had a liver or lung transplant without talking with your doctor.
Do not take Rapamune if you know you are allergic to sirolimus or any of the other ingredients in Rapamune. Symptoms of an allergic reaction include swelling of your face, eyes, or mouth; trouble breathing or wheezing; throat tightness; chest pain or tightness; feeling dizzy or faint; and rash or peeling of your skin.
Before taking Rapamune, tell your doctor if you have liver problems, skin cancer or it runs in your family, high cholesterol or triglycerides, are breastfeeding or plan to breastfeed, and are pregnant or plan to become pregnant. Women of childbearing potential should use effective birth control before therapy, during therapy, and for 12 weeks after Rapamune therapy has been stopped. Rapamune may interact with other medicines. Make sure that your doctor is aware of all prescription and over-the-counter drugs that you are taking, including vitamins, herbs, and nutritional supplements.
Rapamune may cause swelling in your hands, feet, and in various tissues of your body. Call your doctor if you have trouble breathing.
Rapamune may cause your wounds to heal slowly or not heal well resulting in redness, drainage, or opening of the wound.
Rapamune may increase the levels of cholesterol and triglycerides (lipids or fat) in your blood. Your doctor should do blood tests to check your lipids during treatment with Rapamune. Your doctor may recommend treatment if your lipid levels become too high. Your lipid levels may remain high even if you follow your prescribed treatment plan.
In patients taking Rapamune with cyclosporine, decreased kidney function has been observed. Your doctor will regularly check your kidney function.
Rapamune may increase protein in your urine. Your doctor may monitor you for abnormal protein in your urine from time to time.
Rapamune may increase your risk for viral infections. Certain viruses can live in your body and cause active infections when your immune system is weak. One of these viruses, BK virus, can affect how your kidney works and cause your transplanted kidney to fail. A certain virus can cause a rare serious brain infection called Progressive Multifocal Leukoencephalopathy causing death or severe disability.
Rapamune may cause potentially life-threatening lung or breathing problems. Symptoms may include coughing, shortness of breath, or difficulty breathing.
When Rapamune is taken with cyclosporine or tacrolimus, you may develop a blood clotting problem resulting in unexplained bleeding or bruising.
Common side effects associated with Rapamune include high blood pressure, pain (including stomach and joint pain), diarrhea, headache, fever, urinary tract infection, low red blood cell count (anemia), nausea, and low platelet count (cells that help blood to clot). If you experience any side effects, contact your doctor.
Patients should always ask their doctors for medical advice about adverse events.
You may report an adverse event related to Pfizer products by calling 1-800-438-1985 (US only). If you prefer, you may contact the U.S. Food and Drug Administration (FDA) directly. The FDA has established a reporting service known as MedWatch where health care professionals and consumers can report serious problems they suspect may be associated with the drugs and medical devices they prescribe, dispense, or use. Visit MedWatch or call 1-800-FDA-1088.