AstraZeneca and MSD’s Selumetinib Receive MHLW’s Orphan Drug Designation for Neurofibromatosis Type 1 in Japan

AstraZeneca and MSD’s Selumetinib Receive MHLW’s Orphan Drug Designation for Neurofibromatosis Type 1 in Japan

Shots:

  • The ODD designation follows P-I/II SPRINT Stratum 1 study assessing Selumetinib (bid) as monothx. in pediatric patients with NF1-related inoperable PNs
  • The P-I/II SPRINT Stratum 1 study demonstrated a reduction in the size of tumors with 66% ORR in children. AstraZeneca and MSD are co-developing and co-commercializing selumetinib which received the US FDA’s approval in Apr’2020 under the brand name “Koselugo”
  • Selumetinib is a MEK1/2 inhibitor and has received the US FDA’s BT & RPD designation in 2019, ODD in 2018, and EU and Swissmedic’s ODD in 2018 for pediatric patients with NF1 PN. The MAA for the therapy is under EMA’s reviews with other ongoing global regulatory submissions

Click here to read full press release/ article | Ref: AstraZeneca | Image: Business Journal