GSK/Prosensa confirm orphan drug setback
pharmafile | September 23, 2013 | News story | Sales and Marketing | GSK, MD, Prosensa, drisapersen
An experimental treatment by GSK and Prosensa for a rare type of muscle-degenerating disease has failed to meet its primary endpoint in a late-stage trial.
Designed to tackle Duchenne muscular dystrophy (DMD), Drisapersen was found to have no significant impact on physical mobility compared to placebo.
The drug has orphan drug status in the EU, US and Japan, meaning regulations surrounding its development are eased due to the rarity or severity of its target condition.
In June this year, it also was granted ‘breakthrough therapy’ designation by the FDA, further accelerating its development and review in the US.
This Phase III failure represents a major disappointment for GSK, which obtained drisapersen’s commercial rights from Dutch biotech Prosensa four years ago in a deal worth up to £428 million.
The news marks the British company’s second high-profile late stage failure this month, after cancer vaccine MAGE-A3 fell at the last post three weeks ago.
As for Prosensa, its market value plummeted by $600 million (or 70%) after the news was announced, according to Reuters.
The news agency also quotes an analyst at JP Morgan who says a regulatory filing for drisapersen is now ‘very unlikely’.
DMD is a debilitating neuromuscular disease that affects male children. It leads to a gradual loss of muscle function, and claims most patients’ lives before they reach 30.
The condition is caused by a defect in the dystrophin gene. In 13% of cases, this flaw prevents genetic code being read past a particular point. Drisapersen is designed to bypass this problematic code sequence in a process called ‘exon skipping’.
The news of its Phase III failure will be treated cautiously by rival Sarepta, whose drug eteplirsen is also vying to become the first approved treatment for DMD.
The setback for its competitors could be seen as welcome news, but then drisapersen’s poor performance raises further questions about the effectiveness of exon skipping.
GSK and Prosensa’s trial is part of a larger development programme, consisting of six studies in total. Research will continue, with further results expected later this month.
Prosensa chief executive Hans Schikan, said: “While we are disappointed that this study did not meet its primary endpoint, we remain committed to the overall programme.”
He added: “With no long-term disease modifying therapies available for DMD patients, research and development of possible treatment options is of critical importance for boys and their families affected by this debilitating disease.”
Hugh McCafferty
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