HPBCD treatment for Niemann-Pick C: Mallinckrodt communicated results of clinical trial

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Mallinckrodt Pharmaceuticals recently revealed findings of their completed registration trial for VTS-270 (Hydroxypropyl Betadex- HPBCD), a development product to treat Niemann-Pick Type C. In the trial, the product did not show a statistically significant separation from placebo, but importantly, neither the VTS-270, nor the placebo arm showed disease progression – as would have been anticipated in a neurodegenerative condition over 52 weeks of observation. The expectation was that both treatment groups in the study would worsen over the trial period but that the VTS-270 treated group might show an attenuated course based on an accrued benefit. The drug was given by spinal injection into the cerebrospinal fluid, which circulates to the brain.

Nevertheless, all patients who participated and who wish to are continuing to receive drug injections, in the next “open-label” stage of the trial. The study sponsor – regardless of the discouraging data – remain deeply committed to the research project.  In the coming months Mallinckrodt will continue to work with the primary investigators and the FDA to clarify a potential path forward.

The press release of Mallinckrodt was referred in the online platform of Science magazine.




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