Here, Lucinda Cash-Gibson and Francesco Patalano discuss why the pharmaceutical industry is collaborating to develop protocols for patient-centric integrated platform trials and what the clinical research industry may look like in future.
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Neurofibromatosis type 1 (NF1)
The European approval of Koselugo was based on its ability to reduce inoperable plexiform neurofibromas in paediatric patients with neurofibromatosis type 1.
The Committee for Medicinal Products for Human Use (CHMP) recommended eight drugs for approval and nine indication extensions.
Forty percent of neurofibromatosis type 1 patients enrolled in the trial had a >20 percent reduction in tumour burden with cabozantinib.