Boehringer drug lands US, EU orphan tag

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EMA advisory panel backs Lilly's empagliflozin
Boehringer drug lands US, EU orphan tag

Boehringer Ingelheim announced Thursday that its experimental acute myeloid leukemia (AML) medication volasertib has joined the FDA and European Medicines Agency's rosters of drugs with an “orphan” status.

The PLK-1 inhibitor works by tamping down on high rates of cell division, a characteristic of AML, which has one of the lowest survival rates among leukemias. The orphan status is in addition to an FDA Breakthrough Therapy designation the drugmaker won for the medication earlier this year, which will expedite the review process for the rare disease therapy.

The two-tag status hits an FDA sweet spot of sorts: the IMS Institute for Healthcare Informatics noted in a recent report that last year's pharmaceutical launches “were strengthened by a high number of orphan approvals and the increase in applications and approvals for drugs seeking the new FDA Breakthrough Therapy Designation.” IMS also noted that 17 drugs with orphan status launched in 2013, bringing the five-year total to 53, compared to 2008 when that total stood at 29.

The cancer spreads quickly if it is not treated early, and is the most common type of adult acute leukemia.

The American Cancer Society puts the average age of an AML patient at 66, occurring slightly more among men than women.

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