POMPE - Alpha-Glucosidase, Birth of a new medicine.
Pharming is aiming to develop a less immunogenic GAA
from its transgenic rabbit platform. than Myozyme/Lumizyme.
The product will not be considered a ‘Biosimilar’ by the authorities
as it is produced on a totally different production platform,
but from an activity and safety perspective,
this new product will be broadly biosimilar to Myozyme/Lumizyme.
The approach by Pharming (if successful) may also result in a so-called ‘Biobetter’.
In 2019, sales of Myozyme®/Lumizyme® were EUR 918 million,
an increase of 8.3%.
On this basis, assuming a similar growth for the products in 2020,
the size of the Pompe disease market globally may be estimated
at approximately EUR 1-1.3 billion.
Recently, Pharming indicated that it plans to initiate
a Phase I/II trial in Pompe Disease in the beginning of 2021.