Results from Phase II/III Trial Bring Hope for FDA Approval for New Niemann-Pick Disease Type C Treatment

Niemann-Pick Disease is a genetic lysosomal storage disease. Lipids accumulate in the lysosomes of cells, causing debilitating symptoms. There are three forms of the disease- A, B, and C. Each form has its own unique characteristics and symptoms. Niemann-Pick Disease Type C (NPC) is caused by mutations in the NPC protein. This form of the disease affects approximately 1,000 to 2,000 people in the United States and Europe. Europe has one approved treatment for the condition but there remain to be no approved therapeutic options for NPC in the United States.

But a recent clinical trial by Orphazyme has shown promise for the development of a new treatment for this condition in the U.S and Europe.

The Trial

Orphazyme has announced the completion of their Phase II/III trial examining orally administered arimoclomol as a treatment for NPC. This study was conducted at multiple centers across the U.S. and the EU over 12 months. It included 50 participants who were between 2 and 18 years of age. These patients were randomized at a scale of 2:1 to receive either arimoclomol or placebo. The trial aimed to assess the drug’s safety and efficacy for NPC. The drug was administered in addition to routine care.

Arimoclomol has already been granted Rare Pediatric Disease, Orphan Drug, and Fast Track designations by the FDA for Niemann-Pick Disease Type C. It works by increasing production of HSPs which helps to improve the function of the body’s lysosomes. In addition to being evaluated for NPC, researchers are studying its effect in Gaucher Disease, Amyotrophic Lateral Sclerosis, and sIBM.

Here is a summary of some of the most notable results from this Phase II/III trial-

  • Arimoclomol showed a 74% reduction in disease progression.
  • Only 10.7% of those taking arimoclomol severely progressed in the disease versus 26.7% who received placebo.
  • Similar adverse events were documented for both the arimoclomol (88.2%) and placebo (81.3%) groups. Serious adverse events were 14.7% and 37.5% respectively.
  • Those treated with arimoclomol showed reductions in disease-related lipids which were statistically significant.

Researchers are extremely encouraged by these results and are confident in arimoclomol’s ability to provide significant clinical benefits to patients with NPC. This trial also shows that arimoclomol has significant potential as a treatment for other protein-misfolding conditions.

Looking Forward

In addition to the results from this trial, Orphazyme expects 24 months worth of data from their ongoing extension study of the drug to be available by the third quarter of this year.

Orphazyme is beginning preparations to file their drug applications for arimoclomol to the FDA and EMA approval. They state their goal is to get the drug into the hands of patients as soon as possible. They will meet with these organizations in mid-2019 and their estimated timeline for arimoclomol’s potential approval is 2020.

Researchers at Orphazyme say they understand the need NPC patients have to obtain a better therapeutic option quickly. Hopefully, we will see arimoclomol become accessible to patients with NPC in the near future.

You can read more about this Phase II/III trial and Orphazyme’s work on arimoclomol here.


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