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Hyperion获得FDA批准新药Ravicti

  1. Hyperion
  2. Ravicti
  3. 尿素循环紊乱

来源:生物谷 2013-02-04 21:31

Hyperion公司开发新药首次获得FDA审核通过,这家位于South San Francisco的公司于2012年向FDA提交了其研发的治疗尿素循环紊乱的新药RAvicti,FDA批准该药用于两岁以上的患者。消息已经披露就引起公司股价上涨。研究表明相对于传统疗法Buphenyl,患者只需要一天三次服用即可,且药效和传统疗法相当。

Hyperion公司开发新药首次获得FDA审核通过,这家位于South San Francisco的公司于2012年向FDA提交了其研发的治疗尿素循环紊乱的新药RAvicti,FDA批准该药用于两岁以上的患者。消息已经披露就引起公司股价上涨。研究表明相对于传统疗法Buphenyl,患者只需要一天三次服用即可,且药效和传统疗法相当。

据报道,尿素循环紊乱是由于体内酶合成基因的缺失造成的,大约10000个新生儿中会有一个患儿。Hyperion公司称,这种新药的通过将为治疗这种疾病提供新的选择,使更多患者受益。(生物谷Bioon.com)

详细英文报道:

Hyperion ($HPTX) has won its first FDA approval. The South San Francisco-based company grabbed the agency's nod for its drug Ravicti, a treatment for managing genetic conditions known as urea cyclic disorders.

The approval comes after U.S. regulators delayed their decision last year on Hyperion's application to market the med for UCDs. Triggering enzyme deficiencies, the disease leads to toxic buildups of ammonia in the blood that can lead to brain damage and coma. The agency green-lighted the drug for patients aged two years or older with the disorders, which afflict one in 10,000 births, helping the company garner orphan status for Ravicti.

Hyperion is likely to compete against an existing therapy on the market for combatting UCDs, Buphenyl. The biotech proved that its new med, which is taken orally as a liquid three times a day with meals, was just as effective as the older remedy. Now the company faces inevitable hurdles from payers, which can be stingy on reimbursing for meds unless they show improved benefits for patients.

"Ravicti provides another treatment for chronic management of urea cycle disorders, a group of life-threatening conditions," said Dr. Donna Griebel, M.D., director of the Division of Gastrointestinal and Inborn Errors Products in the FDA's Center for Drug Evaluation and Research, in her agency's release. "The approval of this new therapeutic option demonstrates FDA's commitment to providing treatments for patients suffering from rare diseases."

The company's shares jumped 3.2% to $16.13 per share as of 12:38 p.m. ET today. Its stock price has shot up 58% since its lackluster IPO in July, when the shares hit the public market below the company's hoped-for range.

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