luding those directly linked to pts’ daily activities, provided only limited supportive evidence. Finally, insufficient data had been provided to determine how the medicine works in the body and how its effects change with the dose [18].
24/01/2014 15:41:35
Mar 13: Being considered for conditional approval in the EU [16].
09/03/2013 19:09:44
Dec 12: EMA accepts filing for ataluren to treat pts with nonsense mutation Duchenne muscular dystrophy (nmDMD) [15].
10/12/2012 09:32:11
Jul 12: Granted orphan drug status in the EU for treatment of Becker muscular dystrophy (EU/3/12/1010) [14].
27/07/2012 12:05:58
PIIb (n=174) data will be the basis of ‘interactions’ with the US FDA & national regulatory authorities in Europe in 4Q 2010 [10].
20/10/2010 11:41:52
April 10: Filing on hold due to failure of PIIb study to meet primary endpoint (9)
12/04/2010 10:44:03
Dec 09: Company hope to file in US mid-2010. Could be approved in 2011 [5].
23/12/2009 09:07:42
In June 2009, PTC Therapeutics received a four-year grant from the FDA’s Office of Orphan Products Development (worth $US1.6 million), to support the pivotal trial of ataluren. The FDA has also granted Subpart E designation for expedited development, eva luation and marketing [3].
13/09/2009 18:28:08
PII/III international study started Apr 08 [3].
13/09/2009 18:27:42
Fast track in US with orphan status. Orphan status in EU (1). PII trials in duchenne muscular dystrophy (2).
Trial or other data
Oct 15: NHS England issued a clinical commissioning policy in July 2015, stating that it will not routinely commission ataluren for patients with nmDMD. Where an individual’s clinician believes that there may be exceptional clinical circumstances that might warrant consideration of funding outside of this policy, an application can be made under NHS England’s Individual Funding Request (IFR) procedure. A review of this policy position will be considered once NICE has published its final guidance [31].
21/10/2015 12:38:21
Oct 15: In DRAFT guidance, NICE is minded not to recommend ataluren for treating DMD with a nonsense mutation in the dystrophin gene, as it has not yet been presented with an adequate justification for its considerable cost. The Highly Specialised Technologies eva luation Committee has recommended that NICE requests further information from the company on the size of benefit provided for patients, carers and family members. NICE also requires further justification for the cost of ataluren per patient (average of £220,256 per person per year), taking into account the size of the benefit after further clarification, and compared with the benefit obtained with other highly specialised technologies available to NHS patients [30].
21/10/2015 12:36:52
Oct 15: PTC Therapeutics announces results from the PIII ACT DMD trial. In the overall intent-to-treat study population, the primary endpoint of change from baseline in 6MWT demonstrated a 15 meter benefit (p=0.213), which was not statistically significant. A highly significant benefit of 47 meters (p=0.007) was demonstrated in the pre-specified patient population of 300-400 meters at baseline as measured by the 6MWT. No patients in this group lost ambulation (0/47) versus four patients in the placebo group (4/52). Translarna showed a benefit over placebo across key se |
