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PTC向欧盟提交囊性纤维化药物Translarna的上市申请(三)
2016-02-02 04:26:28 来源: 作者: 【 】 浏览:3643次 评论:0
condary and tertiary endpoints, including timed function tests (10 meter Run/Walk, 4 Stair Climb, 4 Stair Descend) and the North Star Ambulatory Assessment test. In addition, a pre-specified meta-analysis of the combined placebo-controlled ACT DMD and Phase 2b trials demonstrated a statistically significant benefit of Translarna across the primary (p=0.015) and key secondary endpoints. After completion of ACT DMD, 97% of patients who completed ACT DMD enrolled in the extension study [29].
19/10/2015 09:53:57
Current options (corticosteroids) can delay but not prevent loss of walking ability. Ataluren is the first therapy to target the underlying defect in nmDMD, but will benefit only a small proportion of all DMD patients. It is likely to be additional to current therapy in ambulant patients but could prolong independence and delay complications
01/10/2015 15:01:51
Mar 14: NCT02090959 A PIII extension study in 220 patients with nonsense mutation dystrophinopathy who participated in a previous PIII study of ataluren to eva luate long term safety.Patients will receive ataluren for 96 weeks. The study starts Mar 14 and is due to complete Jun 17 [21].
21/03/2014 08:53:30
Apr 13: NCT01826487 is a PIII efficacy and safety study of ataluren in 220 patients with nonsense mutation dystrophinopathy. Subjects will be randomized in a 1:1 ratio to ataluren 10-, 10-, 20-mg/kg dose level or placebo. The primary outcome is change in the distance walked during a 6-minute walk test from baseline to 48 weeks. The study started Mar 13 and is due to complete Jun 15. An open-label extension study is planned for patients who successfully complete the double-blind study in countries where ataluren is not commercially available [17]
09/04/2013 09:09:06
Mar 13: PTC plans to start a PIII confirmatory study in H1 2013 involving 220 nmDMD patients, with the 6-minute walking distance test as the primary outcome [16]. 
09/03/2013 19:09:59
Jun 12: NCT01247207 (US based, n=110) and NCT01557400 (multicentre including Europe, n=96) are two open-label PIII extension studies of patients with nonsense mutation dystrophinopathy who received ataluren in a prior PTC-sponsored study. The primary objective is to eva luate the long-term safety of ataluren, as determined by adverse events and laboratory abnormalities. The US study is due to complete Oct 12, and the other, which started May 12,is due to complete in May 13 [13]
07/06/2012 14:59:56
Sep 11: PTC and Genzyme have restructured their collaboration. Under the original agreement, commercial rights were held by PTC for the US and Canada and by Genzyme in all other countries. PTC has now regained worldwide rights to ataluren and Genzyme retains an option to commercialize ataluren in indications other than nonsense mutation Duchenne/Becker muscular dystrophy outside the US and Canada [11]. 
07/06/2012 11:07:55
Nov 10: A PIII, open-label study (PTC124-GD-016 DMD; NCT01247207) to determine the long-term safety of ataluren in previously-treated pts with nonsense mutation Duchenne/Becker muscular dystrophy began recruiting approx 110 boys at sites in the US. The study is due to complete collection for the primary outcome measures of safety & tolerability in Jul 12 [12].
07/06/2012 11:05:18
Oct 10: data for the PIIb trial (n=174) (see below) for ataluren in the treatment of pts with nonsense mutation dystrophinopathy (nmDBMD; comprising Duchenne and Becker muscular dystrophy), presented at the Interna
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