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A survey of breakthrough therapy designations

Nature Biotechnology volume 32, pages 323330 (2014) | Download Citation

As of last month, 41 products have been granted breakthrough therapy designations by the US Food and Drug Administration—drugs against cancer, hepatitis C and monogenetic diseases predominate.

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    Phase I/II study of volasertib (BI 6727), an intravenous Polo-like kinase (Plk) inhibitor, in patients with acute myeloid leukemia (AML): results from the randomized phase II part for volasertib in combination with low-dose cytarabine (LDAC) versus LDAC monotherapy in patients with previously untreated AML ineligible for intensive treatment. Oral presentation at ASH Annual Meeting and Exposition, New Orleans. (8–11 December 2012).

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    Final data from phase 2 combination study of VX-809 and KALYDECO (ivacaftor) showed statistically significant improvements in lung function in people with cystic fibrosis who have two copies of the F508del mutation.

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    BioMarin initiates phase 3 trial for amifampridine phosphate for the treatment of LEMS (7 June 2011).

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    Portola pharmaceuticals announces new phase 2 results confirming immediate, dose-dependent and well-tolerated reversal of anticoagulation activity of XARELTO(R) (rivaroxaban) with andexanet alfa (PRT4445*), investigational Factor Xa inhibitor reversal agent. (9 December 2013)

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The author thanks Novel Health Strategies, the Institute for Global Policy Research, the American Society of Clinical Oncology, the American College of Rheumatology, the American Association of Liver Diseases, American Heart Association, the American Society of Hematology and the Biotechnology Industry Association for providing support for this article.

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  1. Saurabh (Rob) Aggarwal is at Novel Health Strategies LLC, Bethesda, Maryland, USA, and the Institute for Global Policy Research, Washington, DC, USA.

    • Saurabh (Rob) Aggarwal


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Correspondence to Saurabh (Rob) Aggarwal.

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