Pink Sheet’s Drug Review Profile follows the FDA’s navigation of the first expanded umbilical cord blood graft with the first indication grounded in neutrophil recovery and infection incidence data.

South Korean firm teams with Speragen to develop enzyme-replacement therapy for the genetic disorder and with Mirum for maralixibat. Plus deals involving Takeda/PeptiDream, Hanmi/LegoChem, Sun/Cassiopea, LegoChem/Cellectar, ChubLab/CJ CheilJedang Corp., Alebund/Chugai, Kintor/Fosun, Astellas/ExCellThera and I-Mab/neoX/Immorna.

Last year saw the number of applications accepted onto the European Medicines Agency’s priority medicines scheme inch up and the notoriously high rejection rate for applications drop.

Regulatory review resource-intensive cell and gene therapy licensing applications are rolling toward US FDA along expedited pathways, while complete response letters and missed goal dates start to stack up under pandemic inspection constraints.