Continued Access to Drisapersen for Patients Completing the US Ph IIb 48-week study

Below is the latest update PPMD has received from Dr. Rohit Batta, Global Medical Affairs and Patient Relations Leader in GSK's Rare Disease Unit :

The purpose of this communication is provide an update to you, as you have requested, on GSK’s plans for providing the option of continued access to drisapersen for boys completing the US Ph IIb 48 week study (DMD114876) -

GSK is committed to providing boys who have completed the clinical studies with continued access to drisapersen whilst ensuring that appropriate safety monitoring is maintained, as we seek regulatory approval to make the medicine available in that country.

We appreciate the travel burden that boys and their carers face when participating in the clinical studies so we have also been investigating ways in which this may potentially be reduced while providing continued access to this investigational medicine in line with study guidelines. One means of achieving this is through the use of a treatment protocol (rather than a research protocol). This can allow for local laboratory assessments for most parameters and can allow physicians who may be closer to the patient's home to participate, thus sometimes minimizing travel for patients and carers.  GSK has finalized the treatment protocol and now that it has also been through the relevant FDA review, we are engaging with participating sites with the aim of beginning to initiate the treatment protocol in the US by mid-late summer for US citizens.

In the meantime, for boys who have completed the 48 week DMD114876 study prior to the treatment protocol being initiated, a temporary bridging extension protocol is in start-up at DMD114876 sites with the earliest completers - clinical

This extension protocol will not be activated in additional sites due to the proximity of the expected start of the treatment protocol. Patients within the open-label extension study will eventually be offered participation in the treatment protocol which should help reduce the travel burden while providing continued access to this investigational medicine. It is important to note that drisapersen is an investigational medicine that has not been approved by the FDA for any indication.


Other recent updates from GSK

Learn more about drisapersen

Drisapersen (previously GSK2402968) consists of short pieces of DNA called “antisense oligonucleotides” or “AONs” that are being tested for their ability to convert deletions near Exon 51 in the dystrophin gene from non-functional “out-of-frame” deletions to more functional “in-frame” deletions, such as those typically seen in boys and men with Becker muscular dystrophy. The strategy is commonly called “exon-skipping”.


Although variations on this strategy might ultimately be used to try to correct deletions in many parts of the dystrophin gene, GSK 2402968 targets the following deletions: 45-50, 47-50, 48-50, 49-50, 50, 52.


 Duchenne Research


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