AstraZeneca says its trial drug for thyroid cancer granted US FDA orphan drug designation

pharmafile | May 12, 2016 | News story | Research and Development, Sales and Marketing AstraZeneca, Orphan Drug Designation, US FDA, drug trial, regulation 

AstraZeneca (LSE: AZN) on Thursday said the US Food and Drug Administration (FDA) has granted Orphan Drug Designation its trial drug to treat late-stage differentiated thyroid cancer.

The company said the drug compound, selumetinib, inhibits a chain of protein cells called MEK pathway, in cancer cells to prevent tumour growth.

The drug is currently in Phase III trials, the company said in a statement.

Sean Bohen chief medical officer, at AstraZeneca, said: “Uptake of RAI is crucial for patients with thyroid cancer where no other therapies have proven beneficial. Selumetinib could significantly enhance currently available treatment options for these patients. The Orphan Drug Designation is an important achievement as we advance our development plans for this potential treatment in differentiated thyroid cancer.”

Differentiated thyroid cancer (DTC) is diagnosed in about 60,000 people in the US each year, and radioactive iodine (RAI) is recommended for those with known/ suspected metastases at diagnosis and those at high risk of recurrence.

The Orphan Drug Designation provides orphan status to drugs and biologics, which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.

The drug compound selumetinib is also in late-stage trials for non-small cell lung cancer.

Anjali Shukla

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