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Selumetinib granted orphan drug designation in Japan for NF1

Jose Baselga

AstraZeneca’s Selumetinib has been granted orphan drug designation (ODD) in Japan for the treatment of neurofibromatosis type 1 (NF1).

Selumetinib is co-developed and co-commercialised with MSD.

“Neurofibromatosis type 1 can have a devastating impact on children and new medicines are urgently needed to help treat the resulting plexiform neurofibromas and associated clinical issues. Current options in most countries are limited and this designation is a significant step forward in bringing the first medicine for NF1 to paediatric patients in Japan,” says José Baselga, Executive Vice President, Oncology R&D, AstraZeneca.

The SPRINT Stratum trial

The Japanese Ministry of Health, Labour and Welfare grants ODD to medicines intended for the treatment of diseases that affect fewer than 50,000 patients in Japan and for which there is a high unmet medical need.

The National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored Phase I/II SPRINT Stratum 1 trial showed an overall response rate (ORR) of 66% (33 of 50 patients, confirmed partial response) in paediatric patients with NF1 PN when treated with selumetinib as a twice-daily oral monotherapy. ORR is defined as the percentage of patients with confirmed complete or partial response of at least 20% reduction in tumor volume.

Joint development and commercializing

AstraZeneca and MSD are jointly developing and commercializing selumetinib which was approved in the US in April 2020 under the medicine name Koselugo for the treatment of paediatric patients two years and older with NF1 and symptomatic, inoperable PN.

A marketing authorization application in NF1 PN was accepted for review by the European Medicines Agency earlier in the year and further global regulatory submissions are underway.

Photo of José Baselga: AstraZeneca