Muscular Dystrophy Campaign
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London
SE1 0BU
61A Great Suffolk Street
London
SE1 0BU
Thursday 25 July 2013
Sarepta
Therapeutics eteplirsen update
In a press release, Sarepta Therapeutics yesterday
announced that they plan to submit a licensing application for eteplirsen early
in 2014. If the application is successful, Sarepta could be given permission to
market eteplirsen in the USA. The company has also announced further details of
a phase 3 trial being planned for next year and given an update on the results of
the phase 2b trial that is ongoing.
In a press release, Sarepta Therapeutics yesterday
announced that they have held recent meetings with the Food and Drug
Administration (FDA), the drug regulator in the USA, in which they presented
the latest results from their ongoing phase 2b trial of eteplirsen. Eteplirsen is a
potential exon skipping drug for boys with Duchenne muscular
dystrophy. Following these meetings, the FDA has said they would be willing to consider
a licensing application based on the current trial results. Sarepta now plans
to submit the application early in 2014.
A
licensing application means that the FDA will review the evidence from clinical
trials and pre-clinical testing of eteplirsen. They will decide whether the
potential drug is safe and effective and decide whether Sarepta should be given
a licence to market eteplirsen in the USA. It must be noted that this
announcement is not a guarantee that the licence will be granted - just that
the FDA is willing to look at an application. Also, the application will only
apply to the USA - a separate application will need to be made in Europe.
Sarepta is now working towards producing the application and will continue to
meet with the FDA to make sure the application is as complete as possible.
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Along
with planning a license application, the company has started to scale up
production of eteplirsen, a process which is so far going according to plan.
Although the scaling up process will require a lot of testing and careful
quality control, Sarepta hopes that by the end of 2014 it could be in a
position to supply eteplirsen to between half and all of the boys in the USA
who could benefit from the potential drug. If a license is not granted by the FDA,
the increased production will be used to support a larger phase 3 trial which
Sarepta is currently planning. This will aim to confirm the results of the
current trial (see below for the latest results) and is likely to include
approximately 50 boys each in a treatment and control group (who will not
receive eteplirsen). The design of the trial is still being finalised and we
will bring you more details when they become available.
Recently, Sarepta also gave an update on the latest
results of their ongoing phase 2b trial of eteplirsen. At a conference in
Massachusetts, the company announced that the distance boys canwalk in six minutes is still stable after 84 weeks. Although this
is encouraging, the trial includes only ten boys in total and so the results
must be viewed with some caution. The company is still performing regular
check-ups on all the boys in the trial to monitor the safety and effectiveness
of the potential drug and plans to present results up to 96 weeks at a meeting
of the World Muscle Society in October.
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