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AZ gets orphan status for selumetinib in thyroid cancer

Drug already in phase III testing for KRAS-mutant advanced NSCLC

AstraZeneca headquarters

AstraZeneca has claimed orphan drug status in the US for selumetinib - one of its second-tier oncology prospects - as a treatment for advanced differentiated thyroid cancer (DTC).

Selumetinib is a MEK 1/2 inhibitor that is already in phase III testing for KRAS-mutant advanced non-small cell lung cancer (NSCLC) and a phase II trial in children and adolescents with neurofibromatosis type 1, a genetic disorder that causes tumours to grow along nerves.

The drug is being developed for the small proportion of patients with DTC who do not respond to treatment with radioactive iodine (RAI) - the recommended therapy for DTC cases that have spread and have a high risk of recurrence.

Non-responders to RAI typically do not express a protein - known as sodium/iodine symporter (NIS) - which allows the iodine to be taken up by thyroid cells. AZ has shown that selumetinib increases the expression of NIS and so could be an add-on treatment for patients who do not respond well to RAI.

Selumetinib is currently being tested in the ASTRA study, a phase III trial which is comparing the complete remission rate following a 5-week course of selumetinib or placebo and single dose adjuvant radioactive iodine therapy in patients with DTC.

There are around 60,000 cases of DTC in the US each year, well below the threshold of 200,000 patients that is used to define orphan status. A drug with orphan status attracts grants, tax incentives and the extension of exclusive marketing rights to a drug by seven years.

Orphan drugs accounted for $54bn in sales in the US last year and are expected to reach $60bn in 2016, according to EvaluatePharma data.

The drug is not one of the top oncology prospects at AZ - a group that includes recently-approved NSCLC therapy Tagrisso (osimertinib), Lynparza (olaparib) for ovarian cancer and PD-L1-targeting immuno-oncology candidate durvalumab (MEDI4736).

AZ had also been developing selumetinib in uveal melanoma, but abandoned that programme last year after the drug failed to meet its objectives in a mid-stage clinical trial. Prior to that some analysts had suggested it could become a $1bn product, but now most peak sales predictions fall below the $500m mark.

Article by
Phil Taylor

13th May 2016

From: Research

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