tional Congress of the World Muscle Society. There was a mean difference of 29.7 metres in the change from baseline for the lower dose of ataluren vs. placebo (p=0.058). There was no difference between the high dose & placebo; the authors state that this is consistent with subsequent analysis of non-clinical data, which suggest a bell-shaped dose response curve as a class effect for drugs that promote nonsense suppression [10].
20/10/2010 11:40:02
Mar 10: ataluren failed a 48 week PIIb trial. The primary endpoint -a change in 6-minute walk distance - did not reach statistical significance according to preliminary results. The randomized, double-blind, placebo-controlled trial enrolled 174 participants at 37 sites in North America, Europe, Australia, and Israel. Participants received either a low dose of ataluren (10mg/kg in the morning, 10mg/kg at midday and 20mg/kg in the evening), a high dose of ataluren (20mg/kg in the morning, 20mg/kg at midday and 40mg/kg in the evening), or placebo [8].
03/03/2010 22:16:59
Jan 10: NCT01009294, a I year PIIa study started Nov 09, is eva luating the safety, pharmacodynamic activity, and pharmacokinetics of ataluren, while assessing the use of several outcome measures of physical, pulmonary, and cardiac function in patients with advanced disease. Approximately 30 boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently are being enrolled in the trial at five sites in the US and at one site in the UK. Enrollment will be stratified to ensure eva luation of ~15 participants who are receiving chronic corticosteroid therapy and of ~15 participants who are not. The study is being funded in part by a $1 million grant from the Muscular Dystrophy Association, and will involve MDA´s five-center DMD Clinical Research Network and a site in the UK [6,7].
25/01/2010 11:12:47
PTC Therapeutics is collaborating with Genzyme in the development and commercialization of ataluren. PTC Therapeutics will market ataluren in the US and Canada, while Genzyme will commercialize the product in other regions of the world [4].
13/09/2009 18:45:01
An international pivotal phase II/III trial (NCT00592553) in 174 patients (in the US, Canada, Australia, EU and Israel) with Duchenne/Becker muscular dystrophy due to a nonsense mutation started April 2008. Patients will receive placebo, or one of two doses of ataluren, three times per day for 48 weeks. This will be followed by an open-label extension study. The primary outcome measure is the total distance walked during a six-minute walk test. Results are expected in 2010 [3].
13/09/2009 18:27:54
Shown to restore production of relevant functional proteins in genertic disorders (1). Interim data from the first 2 cohorts of the 3-cohort trial demonstrated that 28 days of PTC 124 (3 dose levels) was associated with increases in muscle dystrophin expression and reductions in serum creatinine kinase values in at least 50% of eva luable patients. 67% of pts receiving the lower dose level and 50% of pts receiving the medium dose level showed an increase in the expression of dystrophin post-therapy.(2)
Evidence Based eva luations
NICE scope http://www.nice.org.uk/guidance/indevelopment/gid-duchennemusculardystrophy/documents
EPAR http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/002720/WC500171816.pdf&n |
