Authored by Karl Simpson
For patients living with Duchenne Muscular Dystrophy, last year offered great hope but also disappointment. The momentum was building in the development of exciting new therapies and they were looking forward to the promise of meaningful treatments that offered the prospect to modify their disease for some of the specific gene mutations. GSK/Prosensa, Sarepta and also PTC Therapeutics were all in human trials with their respective treatments. Now, in early 2014, Prosensa has reported a phase III trial which failed to reach its end point and the product has been returned to them from GSK. Sarepta was advised by the FDA to continue to conduct a phase III trial which will inevitably delay any new drug reaching the market. Now CHMP (Committee for Medical Products for Human Use) at the European Medicines Agency has given PTC Therapeutics a negative opinion on their marketing authorisation application for conditional approval of ataluren.
This leaves DMD patients and their parents without any real joy from what was such a promising landscape just 12 months ago when we published our report on the DMD market. Part of this report was dedicated to the very considerable challenges around advancing rare diseases therapies to market. We looked at why new innovative regulatory approaches will need to be adopted so that these meaningful treatments can reach patients sooner and that real-world clinical data becomes a more common feature of rare disease drug development.
PTC Therapeutics application for conditional approval is part of this approach, given the regulatory framework which exists today. It would allow them marketing authorisation with ongoing post-marketing commitments. Ariad’s much reported cancer drug, Iclusig, presently has this same status in Europe. PTC Therapeutics, while hopeful of conditional approval, communicated quite clearly to its investors that it was not a high probability outcome for the company but they remain committed to completing the trial for alaturen.
Part of this ongoing pursuit is the announcement of several strategic appointments to the executive management of the company. Robert J. Spiegel is appointed as the company’s Chief Medical Officer after a spell consulting with PTC. They have recruited Murad Husain to Vice President of Global Regulatory Affairs, joining PTC from Savient Pharmaceuticals.
Duchenne patients and their parents will be hoping that this management team can complete a successful Phase III trial and get ataluren to market soon.