beeldscherm schreef op 27 november 2017 09:57:
Based on feedback from the FDA, Pharming expects to submit a BLA
supplement (sBLA) for review in 4Q of 2017.
¦ Takeaway #1 - Faster than expected. We had modeled in another phase
3 trial prior to a prophylaxis sBLA, which could have taken another 12-18
months. Thus, a 4Q17 filing is ~12-18 months ahead of our expectations.
¦ Takeaway #2 - Saves money on R&D. From an expense standpoint, the
ability to avoid another phase 3 trial saves the company ~$7 million - $10
million in R&D costs.
¦ Takeaway #3 - Almost doubles the size of the market. We view the
prophylaxis market as approximately the same size (slightly smaller) as
the acute treatment market. Admittedly, Ruconest already receives off label
use for prophylaxis, but this allows the company to better market Ruconest
versus competitive treatments. Thus, this could increase peak penetration
and shorten the launch curve.