An Open-label, Multicenter, Ascending Dose Study of the Tolerability and Safety of Recombinant Human Acid Sphingomyelinase (rhASM) in Patients With Acid Sphingomyelinase Deficiency (ASMD)
Phase of Trial: Phase I
Latest Information Update: 01 Sep 2016
At a glance
- Drugs Olipudase alfa (Primary)
- Indications Niemann-Pick disease type B
- Focus Adverse reactions
- Sponsors Genzyme Corporation
- 04 Jun 2015 According to a Genzyme Corporation media release, olipudase alfa has been granted Breakthrough Therapy designation by the U.S. FDA for the treatment of Niemann-Pick disease type B. The designation is supported by data from this trial.
- 12 Feb 2015 According to a Genzyme media release, results were presented at the Lysosomal Disease Network's WORLD Symposium 2015.
- 12 Feb 2015 Results were published in a Genzyme media release.