Pipeline updates are highly awaited events in the pharma/biotech sector as they play an important role in deciding whether or not to invest in a particular company. These updates provide information on experimental drugs and at times give an insight into the commercial potential of the candidate once it is successfully developed and commercialized.
Late last week, Prosensa Holding N.V. (RNA) announced that following the commencement of re-dosing of drisapersen in the U.S in patients suffering from Duchenne muscular dystrophy (DMD), the company has extended the re-dosing program in Europe. Prosensa informed that the two sites in the PRO051-02/DMD114673 extension study will be reopened, starting with Belgium.
Currently, Prosensa is preparing to extend re-dosing programs into other European countries and globally for all previously treated drisapersen patients through expanded access, compassionate use or named patient programs.
We remind investors that Prosensa had suffered a major setback in Sep 2013, when a phase III study (DEMAND III, or DMD114044) failed to meet the primary endpoint as drisapersen did not show a statistically significant improvement in the 6 minute walking distance (6MWD) test versus placebo. Subsequently, the longest running study, which had been treating boys with drisapersen for almost four years, was put on hold. However, Prosensa came up with encouraging data from a phase II study (DMD114876 or DEMAND V) in Mar 2014.
Based on positive feedback from the FDA, Prosensa intends to seek U.S approval for drisapersen by the end of the year. The company intends to seek European approval shortly thereafter.
We note that drisapersen enjoys orphan drug status in the EU, Japan and the U.S. and Breakthrough Therapy designation in the U.S. Prosensa has full rights to drisapersen after GlaxoSmithKline (GSK) terminated its agreement for the candidate earlier this year.
We are pleased with the company’s efforts to develop drisapersen for the treatment of DMD. We note that several other companies including Sarepta Therapeutics, Inc. (SRPT) and PTC Therapeutics, Inc. (PTCT) among others are developing therapies targeting the DMD market.
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