2020年4月15日-今天，美国食品药品监督管理局批准了杰尔米托（丝裂霉素凝胶）疗法，这是第一种治疗低级别上尿路尿路上皮癌（UTUC）的疗法。尿路上皮癌是泌尿系统内膜的癌症。

“尽管我们国家强调必须对抗COVID-19，但是癌症患者及其独特需求仍然是FDA的重中之重，” FDA肿瘤学卓越中心主任兼代理主任Richard Pazdur医学博士说。 FDA药品评估与研究中心肿瘤病办公室的总部。 “在这一关键时刻，我们将继续加快肿瘤产品的开发。我们的工作人员将继续与药物开发人员，学术研究人员和患者倡导者进行虚拟会面，以推动对癌症药物，生物制剂和设备的协调审查。”

尽管大多数泌尿道上皮癌发生在膀胱中，但UTUC对应于在肾脏或输尿管内壁（将肾脏与膀胱相连的细长管）中出现的一部分泌尿道上皮癌。 UTUC会阻塞输尿管或肾脏，导致某些患者肿胀，感染和肾功能受损。 UTUC可以发展为低度或高度肿瘤。通常，低度肿瘤不是侵入性的，并且很少从肾脏或输尿管扩散。然而，它们经常复发并且治疗涉及治疗可见的肿瘤并试图保留尿路，因为这些肿瘤在泌尿系统中更可能复发而不是扩散。低级UTUC很少见，但每年在美国影响6,000-8,000名新患者。

Pazdur说：“这是专门针对低度UTUC患者的首个批准，为某些可能需要进行肾结直肠切除术的患者提供了选择。” “由于与上尿路复杂的解剖结构相关的重大治疗挑战，许多患者需要接受根治性手术治疗-通常需要彻底清除受影响的肾脏，输尿管和膀胱袖带。 Jelmyto首次为患者提供了低级UTUC的替代治疗选择。”

耶尔米托（Jelmyto）是一种烷基化药物，这意味着它可以抑制DNA转录为RNA，阻止蛋白质合成，并剥夺癌细胞的增殖能力。 FDA根据涉及71名低级别UTUC患者的临床试验结果批准了Jelmyto。这些患者从未接受过治疗或患有至少有一个可测量的乳头状肿瘤（形状类似于小蘑菇，其茎附着在器官内壁上的肿瘤）的低度非侵入性UTUC复发。患者每周接受一次Jelmyto，共六周，如果评估为完全缓解（乳头状肿瘤完全消失），则每月最多接受11个月。在开始治疗后三个月，通过尿液细胞学检查（检查患者尿液中的异常细胞的检查），输尿管镜检查（检查上尿路）和活检（如果有保证）来评估Jelmyto的疗效。

主要终点是治疗开始后三个月的完全缓解。每周进行六次Jelmyto治疗后，在71位患者中的41位（58％）中发现了完全缓解。在开始治疗后的一年中，每三个月使用尿液细胞学检查，输尿管镜检查和活检（如果需要）评估Jelmyto对完全缓解患者的疗效的持久性。达到完全缓解的19名患者（46％）在12个月大关时继续完全缓解。

服用Jelmyto的患者常见的副作用是输尿管梗阻（输尿管变窄或阻塞，可能由于尿液过多而导致肾脏内积水过多），胁腹疼痛（身体一侧出现疼痛），尿路感染，血尿（尿血），肾功能不全（肾脏无法发挥其设计功能），疲劳，恶心，腹痛，排尿困难（排尿疼痛或排尿困难）和呕吐。

果冻菌会引起严重的副作用，包括输尿管梗阻，胁腹疼痛和尿道炎（尿路感染导致血液中的细菌）。输尿管阻塞的患者可能需要短暂或长期的支架以缓解这种阻塞。 51％的因杰尔梅托（Jelmyto）导致梗阻的患者中，梗阻可能是持久性的，不能解决或不能完全解决。肾小球滤过率（用于检查肾脏运作情况的测试）低于30mL / min的患者应避免Jelmyto。

怀孕的妇女不应服用Jelmyto，因为它可能对发育中的胎儿或新生婴儿造成伤害。 FDA建议医疗保健专业人士告诉育龄女性在Jelmyto治疗期间以及最后一次服药后六个月内使用有效的避孕方法。具有生殖潜力的女性伴侣的男性患者也应在用杰尔米托治疗期间以及最后一次服药后三个月内使用有效避孕方法。

当初步的临床证据表明该药物相对于现有疗法有实质性改善时，FDA授予该申请“优先审查和突破性疗法”称号，该名称可加快旨在治疗严重疾病的药物的开发和审查。 Jelmyto还被授予“快速通道”称号，这可以加快对治疗严重疾病和满足未满足的医疗需求的药物的审查。 Jelmyto获得了“孤儿药”称号，该奖项旨在鼓励和鼓励开发罕见病药物。

FDA批准Jelmyto授予UroGen Pharma，Inc.

资料来源：FDA

发表于：2020年4月

2020年4月15日-今天，美国食品药品监督管理局批准了杰尔米托（丝裂霉素凝胶）疗法，这是第一种治疗低级别上尿路尿路上皮癌（UTUC）的疗法。尿路上皮癌是泌尿系统内膜的癌症。

“尽管我们国家强调必须对抗COVID-19，但是癌症患者及其独特需求仍然是FDA的重中之重，” FDA肿瘤学卓越中心主任兼代理主任Richard Pazdur医学博士说。 FDA药品评估与研究中心肿瘤病办公室的总部。 “在这一关键时刻，我们将继续加快肿瘤产品的开发。我们的工作人员将继续与药物开发人员，学术研究人员和患者倡导者进行虚拟会面，以推动对癌症药物，生物制剂和设备的协调审查。”

尽管大多数泌尿道上皮癌发生在膀胱中，但UTUC对应于在肾脏或输尿管内壁（将肾脏与膀胱相连的细长管）中出现的一部分泌尿道上皮癌。 UTUC会阻塞输尿管或肾脏，导致某些患者肿胀，感染和肾功能受损。 UTUC可以发展为低度或高度肿瘤。通常，低度肿瘤不是侵入性的，并且很少从肾脏或输尿管扩散。然而，它们经常复发并且治疗涉及治疗可见的肿瘤并试图保留尿路，因为这些肿瘤在泌尿系统中更可能复发而不是扩散。低级UTUC很少见，但每年在美国影响6,000-8,000名新患者。

Pazdur说：“这是专门针对低度UTUC患者的首个批准，为某些可能需要进行肾结直肠切除术的患者提供了选择。” “由于与上尿路复杂的解剖结构相关的重大治疗挑战，许多患者需要接受根治性手术治疗-通常需要彻底清除受影响的肾脏，输尿管和膀胱袖带。 Jelmyto首次为患者提供了低级UTUC的替代治疗选择。”

耶尔米托（Jelmyto）是一种烷基化药物，这意味着它可以抑制DNA转录为RNA，阻止蛋白质合成，并剥夺癌细胞的增殖能力。 FDA根据涉及71名低级别UTUC患者的临床试验结果批准了Jelmyto。这些患者从未接受过治疗或患有至少有一个可测量的乳头状肿瘤（形状类似于小蘑菇，其茎附着在器官内壁上的肿瘤）的低度非侵入性UTUC复发。患者每周接受一次Jelmyto，共六周，如果评估为完全缓解（乳头状肿瘤完全消失），则每月最多接受11个月。在开始治疗后三个月，通过尿液细胞学检查（检查患者尿液中的异常细胞的检查），输尿管镜检查（检查上尿路）和活检（如果有保证）来评估Jelmyto的疗效。

主要终点是治疗开始后三个月的完全缓解。每周进行六次Jelmyto治疗后，在71位患者中的41位（58％）中发现了完全缓解。在开始治疗后的一年中，每三个月使用尿液细胞学检查，输尿管镜检查和活检（如果需要）评估Jelmyto对完全缓解患者的疗效的持久性。达到完全缓解的19名患者（46％）在12个月大关时继续完全缓解。

服用Jelmyto的患者常见的副作用是输尿管梗阻（输尿管变窄或阻塞，可能由于尿液过多而导致肾脏内积水过多），胁腹疼痛（身体一侧出现疼痛），尿路感染，血尿（尿血），肾功能不全（肾脏无法发挥其设计功能），疲劳，恶心，腹痛，排尿困难（排尿疼痛或排尿困难）和呕吐。

果冻菌会引起严重的副作用，包括输尿管梗阻，胁腹疼痛和尿道炎（尿路感染导致血液中的细菌）。输尿管阻塞的患者可能需要短暂或长期的支架以缓解这种阻塞。 51％的因杰尔梅托（Jelmyto）导致梗阻的患者中，梗阻可能是持久性的，不能解决或不能完全解决。肾小球滤过率（用于检查肾脏运作情况的测试）低于30mL / min的患者应避免Jelmyto。

怀孕的妇女不应服用Jelmyto，因为它可能对发育中的胎儿或新生婴儿造成伤害。 FDA建议医疗保健专业人士告诉育龄女性在Jelmyto治疗期间以及最后一次服药后六个月内使用有效的避孕方法。具有生殖潜力的女性伴侣的男性患者也应在用杰尔米托治疗期间以及最后一次服药后三个月内使用有效避孕方法。

当初步的临床证据表明该药物相对于现有疗法有实质性改善时，FDA授予该申请“优先审查和突破性疗法”称号，该名称可加快旨在治疗严重疾病的药物的开发和审查。 Jelmyto还被授予“快速通道”称号，这可以加快对治疗严重疾病和满足未满足的医疗需求的药物的审查。 Jelmyto获得了“孤儿药”称号，该奖项旨在鼓励和鼓励开发罕见病药物。

FDA批准Jelmyto授予UroGen Pharma，Inc.

资料来源：FDA

发表于：2020年4月

Company: UroGen Pharma Ltd.
Date of Approval: April 15, 2020
Treatment for: Urothelial Carcinoma

Jelmyto (mitomycin gel) is a novel formulation of the approved alkylating drug mitomycin for the non-surgical treatment of patients with low-grade upper tract urothelial cancer (LG-UTUC).

April 15, 2020 -- Today, the U.S. Food and Drug Administration approved Jelmyto (mitomycin gel), the first therapy to treat low-grade upper tract urothelial cancer (UTUC). Urothelial cancer is a cancer of the lining of the urinary system.

“Although our nation’s emphasis is on the need to combat COVID-19, patients with cancer and their unique needs continue to be a top priority for the FDA,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “We continue to expedite oncology product development in this critical time. Our staff is continuing to meet virtually with drug developers, academic investigators and patient advocates to push forward the coordinated review of drugs, biologics and devices for cancer.”

While the majority of urothelial cancers occur in the bladder, UTUC corresponds to a subset of urothelial cancers that arise in the lining of the kidney or the ureter (the long, thin tube that connects that kidney to the bladder). UTUC can block the ureter or kidney, causing swelling, infections and impairment of kidney function in some patients. UTUCs can develop as low-grade or high-grade tumors. In general, low-grade tumors are not invasive and very rarely spread from the kidney or ureter. However, they often recur and management involves treating visible tumors and trying to preserve the urinary tract, as these tumors are more likely to recur in the urinary system than they are to spread. Low-grade UTUC is rare, but affects 6,000-8,000 new patients in the United States every year.

“This is the first approval specifically for patients with low-grade UTUC and provides an option for some patients who may otherwise require a nephroureterectomy,” said Pazdur. “Due to substantial treatment challenges associated with the complex anatomy of the upper urinary tract, many patients need to be treated with radical surgery – usually complete removal of the affected kidney, ureter and bladder cuff. Jelmyto gives patients, for the first time, an alternative treatment option for low-grade UTUC.”

Jelmyto is an alkylating drug, meaning it inhibits the transcription of DNA into RNA, stopping protein synthesis and taking away the cancer cell’s ability to multiply. The FDA approved Jelmyto based on the results of a clinical trial involving 71 patients with low-grade UTUC. These patients had never undergone treatment or had recurrent low-grade non-invasive UTUC with at least one measurable papillary tumor (a tumor shaped like a small mushroom with its stem attached to the inner lining of an organ). Patients received Jelmyto once a week for six weeks and, if assessed as a complete response (complete disappearance of the papillary tumor), monthly for up to 11 additional months. Efficacy of Jelmyto was evaluated using urine cytology (a test to look for abnormal cells in a patient’s urine), ureteroscopy (an examination of the upper urinary tract) and biopsy (if warranted) three months following the initiation of therapy.

The primary endpoint was complete response at three months following initiation of therapy. A complete response was found in 41 of the 71 patients (58%) following six treatments of Jelmyto administered weekly. Durability of the effect of Jelmyto in patients with a complete response was also evaluated using urine cytology, ureteroscopy and biopsy (if warranted) every three months for a year following the initiation of therapy. Nineteen patients (46%) who achieved a complete response continued to have a complete response at the 12-month mark.

Common side effects for patients taking Jelmyto were ureteric obstruction (narrowing or blockage of the ureter that may lead to excess fluid in the kidney due to a backup of urine), flank pain (pain occurring on the side of the body), urinary tract infection, hematuria (blood in the urine), renal dysfunction (inability of the kidney to function in its designed capacity), fatigue, nausea, abdominal pain, dysuria (painful or difficult urination) and vomiting.

Jelmyto can cause serious side effects including ureteric obstruction, flank pain and urosepsis (bacteria in the bloodstream from a urinary tract infection). Patients with ureteric obstruction may require transient or long-term stents to relieve this obstruction. The obstruction may be persistent and did not resolve or did not resolve completely in 51% of patients who experienced obstruction due to Jelmyto. Jelmyto should be avoided in patients with a glomerular filtration rate (a test used to check how well the kidneys are working) of less than 30mL/min.

Women who are pregnant should not take Jelmyto because it may cause harm to a developing fetus or newborn baby. The FDA advises health care professionals to tell females of reproductive age to use effective contraception during treatment with Jelmyto, and for six months following the last dose. Male patients with female partners of reproductive potential should also use effective contraception during treatment with Jelmyto and for three months following the last dose.

The FDA granted this application Priority Review and Breakthrough Therapy designation, which expedites the development and review of drugs that are intended to treat a serious condition, when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Jelmyto was also granted Fast Track designation, which expedites the review of drugs to treat serious conditions and fill an unmet medical need. Jelmyto received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted approval of Jelmyto to UroGen Pharma, Inc.

Source: FDA

Posted: April 2020