CHOLBAM (CHOLIC ACID)CAPSULE Orphacol ( KOLBAM) - France[法国药品]

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CHOLBAM (CHOLIC ACID)CAPSULE Orphacol ( KOLBAM)

2015-03-22 09:54:50 来源: 作者: 【大中小】浏览:2084次评论:0条

胆酸胶囊 CHOLBAM (CHOLIC ACID)CAPSULE Orphacol ( KOLBAM)
美国食品和药物管理局(FDA)批准胆酸胶囊(cholbam，Asklepion制药)用来治疗两种罕见的代谢障碍疾病，这两种代谢障碍疾病阻碍机体产酸，并可能会导致危及生命的肝损伤。
胆酸胶囊可用于以下人群：3周以上婴幼儿，由于单酶缺陷或者过氧化物酶缺陷而导致胆汁酸合成障碍的成年人群。过氧化物酶有助于机体新陈代谢，帮助产生胆汁酸、胆固醇和缩醛磷脂。过氧化物酶缺陷疾病包括齐薇格谱系障碍(Zellweger spectrum disorders)，如齐薇格综合征(Zellweger syndrome)，这种疾病主要表现为异常的面部特征、肝脏肿大、婴儿期的神经损伤。
患有罕见先天性疾病患者缺乏合成胆酸(初级胆汁酸)的酶。胆酸的缺乏可导致胆汁流量减少，有毒的胆汁酸中间体在肝内聚集，食物中的脂肪和脂溶性维生素吸收障碍。
FDA批准胆酸胶囊治疗罕见儿科疾病。虽然被批准，制造商必须对药物的长期安全性进行观察研究。
“这次批准强调了机构要确保对于治疗罕见疾病有效性，” Julie Beitz(FDA药品评价与研究中心的药物评估第三办公室主任)说，“在此药物被批准之前，罕见胆汁酸合成障碍患者没有批准的治疗方案。”
该机构表明，胆酸胶囊治疗由于单酶缺陷导致的胆汁酸合成障碍的有效性是基于一个涉及50例患者的18年的治疗研究。根据大约2/3患者的数据可以评估肝功能的好转。
胆酸胶囊治疗过氧化物酶体缺陷疾病的研究，有29个患者参与，历经18年。46%的患者肝功能好转。
腹泻是在试验中观察到的最常见的不良事件。
FDA表示：经验丰富的肝病学专家或儿科胃肠病学家应监控胆酸胶囊使用，如果患者的肝功能恶化应立即停止使用。

Drug Name(s)	CHOLBAM
FDA Application No.	(NDA) 205750
Active Ingredient(s)	CHOLIC ACID
Company	ASKLEPION PHARMACEUTICALS LLC
Original Approval or Tentative Approval Date	March 17, 2015
Chemical Type	1 New molecular entity (NME)
Review Classification	P Priority review drug

Drug Name	Active Ingredients	Strength	Dosage Form/Route	Marketing Status	RLD	TE Code
CHOLBAM	CHOLIC ACID	10 TO 15 MG/KB	CAPSULE;ORAL	Prescription	TBD ¹⁰	None

Approval History
NDA 205750
Note: Not all reviews are available in electronic format from FDA.
Older labels are for historical information only, and should not be used for clinical purposes.
Approval dates can only be verified from 1984 to the present.
Click on a column header to re-sort the table:

Action Date	Supplement Number	Approval Type	Letters, Reviews, Labels, Patient Package Insert	Note
03/17/2015	000	Approval	Label (PDF)¹² Letter (PDF)¹³

KOLBAM 250 mg - HartkapselnXL. - 092573 - CATALENT UK PACKAGING LTD, GB-WESTHOUGHTON - ASK PHARMACEUTICALS GMBH, D-MUENCHEN
KOLBAM 50 mg - HartkapselnXL. - 092572 - CATALENT UK PACKAGING LTD, GB-WESTHOUGHTON - ASK PHARMACEUTICALS GMBH, D-MUENCHEN
ORPHACOL 250 mg - HartkapselnXL. - 092420 - AMATSI, F-SAINT GELY DU FESC - LABORATOIRES CTRS, F-BOULOGNE
ORPHACOL 50 mg - HartkapselnXL. - 092419 - AMATSI, F-SAINT GELY DU FESC - LABORATOIRES CTRS, F-BOULOGNE
Product details

Product details for Orphacol
Name	Orphacol
Agency product number	EMEA/H/C/001250
Active substance	cholic acid
International non-proprietary name (INN) or common name	cholic acid
Therapeutic area	Digestive System DiseasesMetabolism, Inborn Errors
Anatomical therapeutic chemical (ATC) code	A05AA03
Additional monitoring	This medicine is under additional monitoring. This means that it is being monitored even more intensively than other medicines. For more information, see medicines under additional monitoring.
Treatment of rare diseases	This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.
Exceptional Circumstances	There were "exceptional circumstances" concerning the approval of this medicine. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicine for which authorisation is being sought, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.

Publication details

Publication details for Orphacol
Marketing-authorisation holder	Laboratoires CTRS
Revision	1
Date of issue of marketing authorisation valid throughout the European Union	12/09/2013

Contact address:

Laboratoire CTRS
69, rue d'Aguesseau
92100 Boulogne-Billancourt
France

Product details

Product details for Kolbam (previously Cholic Acid FGK)
Name	Kolbam (previously Cholic Acid FGK)
Agency product number	EMEA/H/C/002081
Active substance	cholic acid
International non-proprietary name (INN) or common name	cholic acid
Therapeutic area	Metabolism, Inborn Errors
Anatomical therapeutic chemical (ATC) code	A05AA03
Additional monitoring	This medicine is under additional monitoring. This means that it is being monitored even more intensively than other medicines. For more information, see medicines under additional monitoring.
Treatment of rare diseases	This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.
Exceptional Circumstances	There were "exceptional circumstances" concerning the approval of this medicine. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicine for which authorisation is being sought, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.

Publication details

Publication details for Kolbam (previously Cholic Acid FGK)
Marketing-authorisation holder	FGK Representative Service GmbH
Revision	3
Date of issue of marketing authorisation valid throughout the European Union	04/04/2014