beeldscherm schreef op 12 november 2017 12:46:
Alpha-glucosidase for the
treatment of Pompe disease
Human recombinant ?-glucosidase has been produced in
transgenic animals before. Until 2002, Genzyme together
with Pharming generated transgenic rabbits producing
?-glucosidase. Production levels were as high as 8 g/L
(Bijvoet et al. 1998, 1999). The transgenic material was
shown to be active in clinical trials. In 2002 all assets
related to the ?-glucosidase program (animals, constructs,
notebooks, IP, etc.) were transferred to Genzyme under
the Settlement Arrangements of 15 August 2002. Genzyme
then stopped the program, preferring to continue with
the better-understood CHO-cell program which became
Myozyme®, but scaling issues forced it to develop a second
almost identical cell-line version to achieve capacity,
which became Lumizyme®. Pharming’s new product is
intended to have better immunogenicity, safety and efficacy
profiles than Myozyme®/Lumizyme®. The product will
not be considered a ‘Biosimilar’ by the authorities as it is
produced on a totally different production platform. The
approach by Pharming (if successful) may therefore result
in a so-called ‘Biobetter’
. In 2015, sales of Myozyme®/
Lumizyme® were €650 million, an increase of 12.4%.On this basis, assuming a similar growth for the products in
2016, the size of the US Pompe disease market globally may
be estimated at approximately US$730 million. In addition
to lower costs of goods, which allow for a forecast lower
price for the new product as compared to Myozyme®/
Lumizyme®, Pharming is aiming for greater ease of
administration. Pharming believes that a significant market
share can be obtained, even though Genzyme currently
holds 100% of the Pompe market.
nu meen ik ook ergens gelezen te hebben dat de Franse lijn (konijnen) die is overgenomen al konijnenbabietjes hebben die al melk geven met (rh)-a-glucosidase