DMD: GSK Returns Drisapersen Development Rights to Prosensa

Multinational pharmaceutical company GlaxoSmithKline has returned the rights to develop Duchenne muscular dystrophy exon-skipping drug drisapersen to biotechnology company Prosensa

All rights to development of experimental Duchenne MD drug drisapersen now belong to Dutch biotech company Prosensa.
Article Highlights:
  • Drisapersen is a drug designed to treat Duchenne muscular dystrophy in patients who can be helped by coaxing muscle cells to "skip" (ignore) exon (section) 51 of the gene for the dystrophin protein.
  • Although it showed early promise, drisapersen did not show benefit compared to a placebo in a recent phase 3 trial.
  • Prosensa says it remains committed to developing drisapersen and other compounds to treat Duchenne MD and will keep the community informed about details; a webinar is scheduled for Jan. 21, 2014, at 8 a.m. Eastern time.
  • MDA remains committed to developing exon skipping and other treatments for DMD.
by Margaret Wahl on January 13, 2014 - 12:23pm

The rights to development of the experimental Duchenne muscular dystrophy (DMD) drisapersen have now been returned to the drug's original developer, Dutch biotechnology company Prosensa. The rights were formerly jointly held by Prosensa and multinational pharmaceutical company GlaxoSmithKline (GSK).

Prosensa and GSK announced the new agreement in a Jan. 13, 2014, press release, in which Prosensa CEO Hans Schikan assured the community that it plans to continue development of drisapersen and other potential treatments for DMD.

Drisapersen — an "exon-skipping" drug for DMD — is designed to cause cells to "skip," or "ignore," a section of the dystrophin gene known as exon 51 and to make shorter-than-normal, but still functional, dystrophin protein molecules. Dystrophin is the protein that's missing in DMD-affected muscles. It's been estimated that 13 percent of boys with DMD may benefit from skipping dystrophin exon 51.

In September 2013, Prosensa and GSK announced the disappointing news that a phase 3 trial had failed to find a statistically significant difference in walking distance or motor function between DMD-affected participants treated with drisapersen and those treated with a placebo.

Prosensa says it will continue DMD drug development

In the Jan. 13 press release, Prosensa CEO Schikan said: "We are fully committed to our mission of developing innovative, RNA-based therapeutics to address unmet medical needs for patients with rare genetic disorders. Prosensa is now in a favorable strategic position to advance the DMD portfolio, which includes drisapersen and five additional compounds, three of which are currently in clinical development. We will continue to work closely with patient groups, investigators, academia and regulators to ensure that we do everything we can to bring treatments to boys affected by DMD."

He added, "GSK has been a valuable development parnter, and we are grateful for the commitment made by the company over the last four years to develop a disease-modifying therapy for this devastating disease."

GSK says Prosensa will keep community informed

GSK's Global Patient Relations Team released the following Jan. 13 statement to patient groups:

To keep you updated, we would like to share with you a decision that has been taken in relation to drisapersen — GSK has transferred to Prosensa all rights to drisapersen for the treatment of Duchenne muscular dystrophy (DMD), which we had originally acquired in 2009.

Prosensa will now have the full rights to choose to continue the development of each of its DMD programs, including drisapersen. GSK and Prosensa have issued a press release to announce this and I have attached the release for reference. [See Jan. 13, 2014, press release.]

We have notified study investigators of these changes and are recommending that boys and their families currently participating in drisapersen clinical studies contact their local clinical study team. You can find information regarding the clinical studies involving drisapersen, by visiting [and entering "drisapersen" in the search box.]

Prosensa will update the community with their plans for drisapersen as soon as they are in a position to do so. We would like to take a moment to recognize the enormous contribution made by boys and their families through participation in the clinical program.

You may have questions in relation to the above, and if so you should direct your enquiry to Prosensa's patient advocacy team at

Moreover, Prosensa will organize a webinar on Tuesday, Jan. 21, at 2 p.m. CET (8 a.m. EST). A live webcast will be available, and the presentation will be archived for 90 days on the Prosensa site at Events & Presentations.

We are honored for the community to support us in conducting the largest global clinical program in DMD, and we again express our gratitude for the help and support your organizations have provided to families, and thank you for being patient as GSK and Prosensa made these important decisions, where the best interests of the DMD boys have been at the heart of all our efforts.

MDA encouraged by Prosensa's commitment

"We’re disappointed that multinational pharmaceutical company GlaxoSmithKline has decided to end its collaboration with Dutch biotechnology company Prosensa for the development of drisapersen, an experimental drug for Duchenne muscular dystrophy," said Jane Larkindale, MDA’s vice president of research. "However, we’re encouraged by the statement made this morning by Hans Schikan indicating that his company is committed to drisapersen and other compounds for DMD. We at MDA of course remain committed to doing everything possible to bring these and other potential DMD treatments into the clinic."

MDA has funded exon-skipping research since 1997 and continues to fund research in exon skipping for DMD.

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