Prosensa intends to file for approval of its Duchenne muscular dystrophy treatment drisapersen later this year in both Europe and the US after positive feedback from regulators.
The Netherlands-based biotechnology company said it now has a clear regulatory path to follow for drisapersen, its lead drug candidate, and should be able to file for conditional approval based on a commitment to carry out two confirmatory studies once the drug is made available to patients.
Drisapersen’s prospects looked fairly rocky earlier this year when former partner GlaxoSmithKline terminated its collaboration with Prosensa, shortly after posting disappointing phase III data which suggested the drug was able to improve a patient’s walking distance compared to placebo, but not by a statistically significant margin.
“Given the urgent need to find effective therapies for boys afflicted with this devastating disease, we could not be more pleased with this favourable outcome,” said Prosensa’s chief executive Hans Schikan.
“We have been diligently preparing for multiple scenarios since acquiring the rights back from GSK in January and completing our more detailed analysis of the drisapersen dataset,” he added.
Investors also responded warmly to the news, with Prosensa’s shares closing up 13 per cent yesterday.
Drisapersen is designed to induce ‘skipping’ of exon 51 when the dystrophin sequence on DNA is written to RNA, with the aim of producing a truncated but semi-functional form of the dystrophin protein that is abnormal in DMD, restoring some muscle function.
A letter from the FDA to the company suggests that Prosensa would need to carry out a confirmatory historically-controlled trial, as well as a randomised study comparing the therapy to another exon-skipping treatment with a similar mechanism of action but which targets a different exon.
Prosensa is developing two other candidates which skip exons 44 (PRO044) and 45 (PRO045). Both are both in early-stage clinical testing.
The news tops off an encouraging couple of weeks for DMD patients, with s block of new therapies now approaching the registration phase.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) recently recommended approval of PTC Therapeutics’ Translarna (ataluren) for the genetic disorder, while Santhera said it is planning to file for approval of Catena/Raxone (idebenone) -used to support lung function in DMD patients – in the coming months. Meanwhile, Sarepta Therapeutics’ eteplirsen is also scheduled for filing in the US before the end of the year.
Analyst Jan De Kerpel of Belgium’s KBC Securities has suggested drisapersen could be a €1bn ($1.4bn) product by the end of the decade, assuming a price of around €200,000 a year, according to a report from Reuters.