Winstgevend schreef op 14 februari 2019 07:16:
Behalve GRAFT - Cin en Pre-eclampsia komt ook Pompe steeds dichterbij:
Given insights and experience gained with RUCONEST®; a similarly highly
glycosylated protein, 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 2017, sales of
Myozyme®/Lumizyme® were EUR 789 million, an increase of 10.1%. On this basis, assuming a
similar growth for the products in 2018, 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 2019. We believe that, considering the
exisiting safety data, this trial can be concluded within 12 months, followed by a Phase II/III trial in
Wordt een druk 1e kwartaal, maar eerst nog wachten op de 2018 cijfers van 7 maart AS
Ik neem aan dat hierna de analisten upgrades volgen.
P.S. nutteloos om je nog druk te maken over 84 losse centen.
Research Triangle Park, NC - Jan. 22, 2019 – Actus Therapeutics, a privately held portfolio company of Asklepios BioPharmaceutical, Inc. (AskBio), today announced the dosing of the first patient in a phase 1/2 clinical study of ACTUS-101 in patients with Pompe Disease. Pompe Disease is a serious disease associated with high morbidity and often leads to premature death. There are variable rates of disease progression and different ages of onset characterized as infantile-onset and late-onset disease.