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All treatments FDA-approved

Miplyffa (arimoclomol)

The first FDA-approved medicine for NPC, taken with miglustat to help fragile NPC1 protein keep working.

Still image of Miplyffa (arimoclomol)
A still of the idea behind Miplyffa (arimoclomol). The motion below is a simple animation, not a replacement for this picture.

How it works

Miplyffa helps the cell’s rescue crew — heat-shock proteins — stay on duty. Those proteins act like chaperones. They try to fold and protect the damaged NPC1 protein so more of it can reach the lysosome and move cholesterol out.

It is approved together with miglustat for neurological symptoms of NPC in people 2 years and older.

Who it is for

Adults and children 2 years and older, used with miglustat.

What it does not do

It does not rewrite the NPC1 gene or restore a fully healthy protein. It is a treatment, not a cure.

Step by step

What this treatment does

Four short pictures of the idea. Play through, or tap a step. This is a teaching picture, not a medical simulation.

Step 1 of 4 · The problem

NPC1 is there, but it is fragile

In many children the NPC1 protein is made, then falls apart. Cholesterol stays trapped in the lysosome.

Official information

Educational only. Ask an NPC specialist what is right for a specific child or adult. Official sites and FDA pages are the source of prescribing information.