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Orphan status for Stem Cell's leukaemia drug

Tigecycline wins FDA rare disease designation
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US regulators have granted orphan drug status to tigecycline, a cancer drug in development by Stem Cell Therapeutics.

The designation provided by the Food and Drug Administration (FDA) is granted to drugs under review for use in rare diseases and comes with several incentives to encourage companies to develop treatment sin these less lucrative therapy areas.

These incentives include seven years of market exclusivity as well as opportunities for additional funding and expert protocol assistance.

Tigecycline is being investigated by Stem Cell Therapeutics for its use in the treatment of acute myeloid leukaemia (AML), a type of cancer that affects a patient's blood and bone marrow.

There were just under 15,000 new cases of the disease reported in the US in 2013 and more than 10,000 deaths.

Stem Cell Therapeutics' VP of drug development Dr Penka Petrova said: “Orphan drug status significantly enhances the commercial potential of tigecycline in AML, a disease which is notoriously difficult to treat.”

Tigecycline is already available in the US under the name Tygacil to treat bacterial infections. Studies have demonstrated that the drug can target leukaemia cells by inhibiting certain proteins, however, influencing the Stem Cell Therapeutics' decision to expand the approved indications.

The orphan designation in AML gives Stem Cell Therapeutics a boost as it looks to edge ahead of companies with rival products in development to offer patients an alternative to intensive treatment with the chemotherapy cytarabine

These companies include Ambit Biosiences, which is developing quizartinib, and Boehringer Ingelheim, which is looking to expands its new oncology business into AML with volasertib.

Article by
Thomas Meek

5th November 2013

From: Research, Sales



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