FDA新核准治疗急性胸腔症候群、急性复发反覆癫痫、回肠囊袋炎等之孤儿药


  January 11, 2008 — 美国食品药物管理局(FDA)以孤儿药方式核准 A-001用于预防镰刀型红血球疾病病患之急性胸腔症候群;核准JZP-8用于治疗癫痫病患的复发急性反覆癫痫;核准 AST-120 用于治疗回肠囊袋炎。
  
  孤儿药 A-001用于治疗急性胸腔症候群
  11月16日,FDA核准孤儿药A-001 (Anthera Pharmaceuticals, Inc) 用于预防急性胸腔症候群(acute chest syndrome,ACS),此症候群是镰刀型红血球疾病患者的主要致死原因。
  
  ACS是一种镰刀型红血球疾病的肺部并发症,特征是迅速且越来越严重的肺部渗入、呼吸急促、呼吸困难、低血氧以及胸痛;最可能发生于幼童,而青少年以及成人的ACS更严重;需要立即与积极治疗ACS,以预防可能的慢性肺衰竭或死亡。
  
  A-001是血清磷酸脂质分解酶A2抑制剂,磷酸脂质分解酶A2在ACS发生时提高,是一种强力发炎调节物质,此药目前正在进行第2期临床试验 (Investigation of the Modulation of Phospholipase in Acute ChesT Syndrome, or IMPACTS)。
  
  孤儿药JZP-8用于治疗复发急性反覆癫痫
  1月7日,Jazz Pharmaceuticals药厂宣布,FDA核准孤儿药JZP-8用于治疗复发急性反覆癫痫(recurrent acute repetitive seizures,ARS)— 在短时间之内增加癫痫发作频率或严重度。
  
  ARS发生于少数已经接受抗癫痫药物治疗的癫痫病患,ARS会变成癫痫持续状态,一种需要紧急治疗的持续癫痫状态。
  
  JZP-8是一种混合的clonazepam新配方,clonazepam是一种 benzodiazepam,用于小发作变化、运动不能发作以及肌抽跃性发作;对succimides无治疗反应的小发作;以及恐慌症;此药设计成鼻喷雾剂。
  
  孤儿药AST-120用于治疗回肠囊袋炎
  12月19日,FDA核准孤儿药AST-120 (Ocera Therapeutics, Inc) 用于治疗回肠囊袋炎,以手术方式建立的储存粪便内囊部位的发炎现象。
  
  慢性肠道疾病,如溃疡性结肠炎或者家族性大肠瘜肉症的病患,可能需接受手术治疗移除一部份的结肠;为了避免用外部方式收集排泄物,使用一部份的小肠建立一个内囊;回肠肛门囊病患约有32%发生回肠囊袋炎,引起的症状包括腹泻、腹部痉挛和疼痛 、排便频率增加、出血、发烧、脱水以及关节疼痛。
  
  AST-120是一种口服药物,包含有吸附性的球型碳微粒,于日本被核准为延长透析时间和减少慢性肾病患者的尿毒症状;目前正进行治疗轻微到中度瘘管型克隆氏症的第3期临床试验。

New FDA Orphan Drugs for Acute

By Jill Taylor
Medscape Medical News

January 11, 2008 — The US Food and Drug Administration (FDA) has granted orphan drug designation to A-001 for the prevention of acute chest syndrome in patients with sickle cell disease, JZP-8 for the treatment of recurrent acute repetitive seizures in patients with epilepsy, and AST-120 for the treatment of pouchitis.

Orphan Drug A-001 for Acute Chest Syndrome

On November 16, the FDA granted orphan drug designation to A-001 (Anthera Pharmaceuticals, Inc) for the prevention of acute chest syndrome (ACS), the leading cause of death in patients with sickle cell disease.

ACS is a pulmonary complication of sickle cell disease that is characterized by rapidly progressive pulmonary infiltrates, tachypnea, dyspnea, hypoxemia, and chest pain. Although it is more likely to occur in young children, ACS occurring in adolescents and young adults tends to be more severe. Immediate and aggressive treatment of ACS is required to prevent possible chronic pulmonary debilitation or death.

A-001 is an inhibitor of serum secretory phospholipase A2, a potent inflammatory mediator that is elevated in ACS. The drug is currently under evaluation in a phase 2 clinical trial (Investigation of the Modulation of Phospholipase in Acute ChesT Syndrome, or IMPACTS).

Orphan Drug JZP-8 for Recurrent Acute Repetitive Seizures

On January 7, Jazz Pharmaceuticals, Inc, announced that the FDA granted orphan drug designation to its JZP-8 product candidate for the treatment of recurrent acute repetitive seizures (ARS) — clusters of seizures that appear to increase in frequency or severity over a short period of time.

ARS occurs in a small subset of patients with epilepsy, despite a regimen of antiepileptic treatment. Unresolved, ARS can progress to status epilepticus, a continuous state of seizure that requires emergency treatment.

JZP-8 is a novel formulation incorporating clonazepam, a benzodiazepam prescribed for petit mal variant, akinetic, and myoclonic seizures; petit mal seizures unresponsive to succimides; and panic disorder. It is designed for delivery by nasal spray.

Orphan Drug AST-120 for Pouchitis

On December 19, the FDA granted orphan drug designation to AST-120 (Ocera Therapeutics, Inc) for the treatment of pouchitis, the inflammation of a surgically created internal pouch for the storage of stool.

Patients with certain types of chronic bowel disease, such as ulcerative colitis or familial adenomatous polyposis, may undergo surgical treatment in which a portion of their colon is removed. To prevent the need for an external appliance to collect waste, a portion of the small intestine is used to create an internal pouch. Pouchitis occurs in an estimated 32% of patients with an ileoanal pouch, causing symptoms such as diarrhea, abdominal cramps and pain, increased stool frequency, bleeding, fever, dehydration, and joint pain.

AST-120 is an orally administered agent composed of adsorptive, spherical carbon microspheres that has been approved in Japan to delay time to dialysis and reduce uremic symptoms of patients with chronic kidney disease. It is currently being studied in a phase 3 clinical trial for the treatment of mild to moderate fistulizing Crohn's disease.

    
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