An experimental drug developed by AstraZeneca that failed last year as a treatment for a rare cancer of the eye has been awarded special "orphan" status in the US for a type of thyroid cancer.
The UK drugmaker, which was banking on cancer treatments to revive its fortunes following patent expiries of several of its drugs, said today, said the decision showed the potential importance of selumetinib for some patients.
Orphan status is awarded to medicines that hold out promise of significant benefit in the treatment of rare, life-threatening diseases.
The designation offers companies, special development and market exclusivity incentives.
The drug is being tested on patients with advanced differentiated thyroid cancer (DTC) who failed to respond adequately to radioactive iodine.
The drug was also being tested as a treatment for advanced non-small cell lung cancer.
According to analysts, it was less important commercially than AstraZeneca's recently launched cancer drugs Tagrisso and Lynparza, and its experimental product durvalumab.
In a press release AstraZeneca said, "DTC is diagnosed in approximately 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. A small proportion of patients do not benefit from currently available treatment with RAI because they do not express sufficient sodium / iodine symporter (NIS) which is important for RAI uptake into thyroid cells. Selumetinib is being tested for its ability to increase expression of NIS with the potential to add a treatment option for patients who do not respond well to RAI.
Sean Bohen executive vice president, Global Medicines Development and 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.''