Olipudase alfa is suitable for the treatment of children and adults with non-neurological symptoms. Clinical studies in 36 adults and 20 children show that the drug improves lung function and decreases liver and spleen volume. These results indicate a clinically relevant effect according to the CHMP. In addition, there are generally no serious side effects. The most commonly reported adverse reactions are reactions related to infections, infusion reactions and gastrointestinal complaints.
Accumulation of fats
Niemann-Pick disease is an inherited metabolic disease in which the enzyme acid sphingomyelinase (ASM) is missing or not functioning. This leads to an accumulation of fats in lysosomes. The disease is also known as (chronic visceral) deficiency of acidic sphingomyelinase (ASMD). Type A usually manifests itself a few months after birth and type B between childhood and adulthood. Some patients have the mixed form type A/B. People who suffer from this disease usually have a larger liver and spleen and more often pneumonia. Olipudase alfa replaces the enzyme ASM and thus prevents the accumulation of fats.
The National Health Care Institute (ZIN) has indicated that the product is eligible for placement in the parcel lock after a positive recommendation from the CHMP.