Shots:

• The conditional approval is based on P-II SPRINT Stratum 1 trial assessing selumetinib (PO- bid) as monothx. in pediatric patients aged >3yr. with NF1-related inoperable PNs. The trial was sponsored by NCI CTEP- conducted under a research & development agreement b/w NCI & AstraZeneca with additional support from NTAP
• The trial showed a reduction in size of tumors with ORR (66%) in children- reduction in pain- improvement in QoL. Safety & efficacy data of the study with longer follow up will be provided as one of conditional approval
• The therapy is approved in the US & other countries for NF1 & symptomatic- inoperable PN while other regulatory submissions of the therapy are ongoing

Ref: AstraZeneca | Image: New Europe