Thank you for donating!

You can donate using the following services.

Orphazyme Reports Encouraging Arimoclomol Clinical Trial Top-line Data in Niemann-Pick Disease Type C (NP-C)

Posted: 03.10.18


  • Arimoclomol was well-tolerated in patients with the rare disease NPC
  • The NPC Phase II/III trial top-line results show a treatment benefit of arimoclomol over placebo on the primary endpoint 5-domain NPC Clinical Severity Scale (NPC-CSS) (p-value 0.07)
  • Albeit statistical significance of 0.05 was not reached, Orphazyme will engage in dialogue with health authorities in the EU and US to determine the best path towards making arimoclomol available to patients with NPC

Copenhagen, September 28, 2018 – Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced encouraging top-line results for its clinical Phase II/III trial with orally administered arimoclomol for the treatment of patients with Niemann-Pick disease Type C (NPC).


The trial was a multi-center, prospective, double-blinded, placebo-controlled interventional study with a 12-month duration. In total, 50 patients were enrolled in the EU and US. The purpose of the trial was to assess the efficacy and safety of arimoclomol, compared to placebo, in the treatment of NPC, administered in addition to the patient’s standard-of-care. The primary endpoints, 5-domain NPC-CSS and Clinical Global Impression of Improvement (CGI-I), evaluated the treatment difference between the arimoclomol-treated and the placebo group after 12 months of treatment.

Overall, baseline characteristics were well-balanced across treatment arms. Arimoclomol was well-tolerated. The overall incidence of adverse events (AEs) was similar for arimoclomol (85.7%) and placebo (81.3%). Serious AEs occurred less frequently in the arimoclomol group (14.3%) compared to placebo (37.5%). The top-line data demonstrated a 74% reduction in progression on the primary endpoint, corroborated by consistent benefit across sub-populations. Placebo progression rates on the CGI-I were lower than expected impeding the ability to show a positive effect.

The full data set, including biomarker data, will become available and be analyzed in Q4 2018. Furthermore, 24-month data will become available from the on-going open-label extension trial in Q2 2019.

Anders Hinsby, Chief Executive Officer of Orphazyme, said: “We are highly encouraged that the top-line data show a strong positive trend on the 5-domain NPC-CSS. We now look forward to receiving the full analysis of data. In addition, the open-label extension trial will provide data on the clinical benefit of arimoclomol over a longer period of time. We are determined to make arimoclomol available to patients as quickly as possible. We are grateful for the strong support we have received from the patient community, the expert physicians, and especially the participants and their families”.


Orphazyme will engage with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to determine the best path towards making arimoclomol available to those suffering from NPC.


Thomas Blaettler, Chief Medical Officer of Orphazyme, said: “We observed compelling, clinically-relevant trends in favor of arimoclomol in this first trial in NPC. I am encouraged by the 74% reduction in disease progression on the 5-domain NPC-CSS. This is further corroborated by a similar effect on the full scale. Assessing the effect in patients 4 years of age and older (44 out of 50 patients), the effect becomes even more pronounced, with a p-value of 0.027”.

This company announcement does not impact the 2018 financial guidance published in the Annual Report 2017 on March 15, 2018.


Conference call

Orphazyme will be hosting an investor call at which Chief Executive Officer, Anders Hinsby, and Chief Medical Officer, Thomas Blaettler, will be presenting the clinical trial top-line data in NPC. The presentation will be followed by a Q&A session.

The call will be held on: Friday, September 28, 2018 at 10.00 AM CET.

Dial-in details:

  • Denmark: +45 35 15 80 49
  • United Kingdom: +44 (0) 330 336 9128
  • United States: +1 929-477-0448

Event Title: Orphazyme Phase II/III clinical trial top-line data in NPC

Confirmation code: 7938836

The presentation will also be available via webcast:

After the call, the presentation will be available by using the following dial-in details:


  • Denmark: +45 70 14 50 87
  • United Kingdom: +44 (0) 207 660 0134
  • United States: +1 719-457-0820