I encourage family members and patients to comment to the FDA that they realize this isn't exactly true and how they feel and their experience if they were in the trial. It worked for CHTP's Northera and it's not clear if it will help for Sarepta but it may. In any case I am pretty sure that the path will be easier with EMA based on the comments they made in the orphan designation approval....
"In addition, although satisfactory methods of treatment of the condition have been authorised in the European Union, the sponsor has provided sufficient justification for the assumption that the medicinal product containing 5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride may be of significant benefit to those affected by the condition. The sponsor has provided preliminary clinical data that demonstrate an improvement in a relevant parameter when the product is used in combination with tetrabenazine. The Committee considered that this could constitute a clinically relevant advantage."
To some of us this suggests that EMA COMP agrees that it is a benefit and that they see something that might indicate it is disease modifying which would be revolutionary for the field of neurodegeneration.
And its quite possible while we have our eyes off the ball [not sure if you say that over there;-)] a surprise accelerated approval could come out of EMA.
A record number of EMA orphan approvals in 2014
EMA is being more aggressive getting orphan medicines to the public. They also provide scientific and development advice to the sponsors to help them through the process. "Record number of medicines for rare diseases recommended for approval in 2014, Number of medicines with new active substances continues to increase. In 2014, the European Medicines Agency (EMA) recommended the highest number of orphan designated medicines for marketing authorisation in a year. Out of the 82 medicines for human use recommended in 2014, 17 are intended for the treatment of a rare disease, providing therapies for patients who often have only few or no treatment options. ++ Special regulatory pathways were used for these three medicines (conditional marketing authorisation for Translarna and Holoclar, and approval under exceptional circumstances for Scenesse). These mechanisms are in place to potentially speed up market access for medicines that fulfill unmet medical needs but for which comprehensive data cannot be provided at the time of application for a marketing authorisation. +++Overall, the number of medicines containing new active substances continues to increase. One in two medicines, either orphan or non-orphan, recommended for approval in 2014, contains a substance that has never been used in medicines before. These medicines have the potential to treat diseases for which no treatments were previously available or bring added benefit to patients over existing therapies. During the past year, EMA provided more scientific support in the early stages of medicine development. Almost seven out of ten applicants received scientific advice from EMA’s Committee for Medicinal Products for Human Use (CHMP) during the development phase of their medicine and this figure rises to four out of five when it comes to innovative medicines. This is a significant increase compared with 2013 when only half of applicants who had a positive opinion for their medicine had received scientific advice."
http://news.centerwatch.com/2015/01...-rare-disease-medicines-for-approval-in-2014/
(20min delay)