beeldscherm schreef op 14 juni 2018 15:07:
laten we toch eerlijk zijn als Pharming binnen nu en 4 jaar een medicijn tegen Pompe op de markt heeft, wat zou de share-price dan wel niet zijn..
Optimistic about Pompe We think Pharming’s platform has great potential to
circumvent the main problem with current enzyme replacement therapy in Pompe
- the instability of proteins derived from Chinese hamster ovary cell-lines.
Only drug approved so far is an enzyme replacement therapy from Sanofi Pompe is
treated through enzyme replacement therapy (ERT). The only drug to have been approved
so far (in both the U.S. and the EU) is Sanofi’s Myozyme/Lumizyme which is recombinant
human GAA produced from Chinese hamster ovary cells. In 2017 Myozyme/Lumizyme
generated sales of €789m. However, Myozyme/Lumizyme has an unfavourable side-effect
profile. The FDA label carries a black box warning of side effects including life-threatening
anaphylactic reactions, severe hypersensitivity reactions, immune-mediated reactions
presenting as nephrotic syndrome and necrotising skin lesions, as well as risk of
cardiorespiratory failure.