Niemann Pick Type C (NPC) is a rare progressive genetic disorder, characterized by the accumulation of dysfunctional proteins causing lysosomal function impairment. Patients can experience diverse symptoms, from hepatosplenomegaly, to neurological disorders, to respiratory failure. New medicines for NPC are expected to launch in the three major markets (3MM: US, Germany and UK) in the next few years and sales are forecasted to reach over $220 million in 2031, forecasts GlobalData, a leading data and analytics company.

GlobalData’s latest report, “Niemann Pick Type C: Opportunity Assessment and Forecast to 2031,” reveals that the US NPC market especially is expected to change in the near future.

Costanza Alciati, Pharma Analyst at GlobalData, comments: “The market growth will be driven by the introduction of pipeline therapies arimoclomol citrate ER, acetylleucine, Trappsol Cyclo and nizubaglustat. Specifically, arimoclomol citrate ER, a heat-shock protein 70 amplifier, is expected to reach the US market by the end of this year, with a Prescription Drug User Fee Act (PDUFA) action date set for 21 June, and could therefore become the first approved drug for NPC in the US.”

While nizubaglustat, currently in Phase II clinical trials in the US, shares the same mechanism of action of miglustat, the other three pipeline therapies would be introducing new mechanisms of action into the NPC market.

However, the key opinion leaders (KOLs) interviewed by GlobalData do not seem too excited about the current NPC pipeline and remain skeptical that the drugs will prove to be efficacious enough to reach FDA and EMA approval.

Alciati concludes: “It seems that arimoclomol citrate, and possibly other new therapies, will be prescribed as an add-on therapy to miglustat, to achieve the highest efficacy possible. But still, it is not going to be enough to reverse disease progression. NPC patients continue to wait for an efficacious cure, and the current drugs in the pipeline still need to prove that their efficacy is high enough for market approval.”