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What Orphan Drug Receives The 1st FDA Breakthrough Therapy Designation For Oncology ?

February 12, 2013

Per a February 12, 2013 Press Release, Pharmacyclics announces that the company is awarded the 1st two Breakthrough Therapy Designations from the US FDA for their orphan drug Ibrutinib for oncology. Under Section 902 of the July 9, 2012, Food and Drug Administration Safety and Innovation Act (FDASIA) signed by President Obama, the new FDA Breakthrough Therapy Designation is described.

Pharmacyclics identifies the 2 FDA Breakthrough Therapy Designations :

1)  Ibrutinib monotherapy for the treatment of patients with relapsed or refractory Mantle Cell Lymphoma (MCL)

2)  Ibrutinib monotherapy for the treatment of patients with Waldenstrom’s Macroglobulinemia (WM).

Both of the oncology indications are B-cell malignancies. By the end of 2013, Pharmacyclics expects to finalize the MCL indication filing and will provide guidance on the WM filing after additional discussions with the FDA. To date five Phase III trials have been initiated with Ibrutinib and a total of 27 trials are currently registered on Ibrutinib has FDA Orphan Drug Designations (ODDs) for 2 indications :

Row Num   Generic Name   Designation Date   Indication


Ibrutinib 12-03-2012 Mantle Cell Lymphoma (MCL)


Ibrutinib 04-06-2012 Chronic Lymphocytic Leukemia

MCL is an aggressive type of B-cell Non-Hodgkin Lymphoma (NHL) that occurs in older adults.  The disease usually starts in the lymph nodes, but can spread to other tissues. Ibrutinib targets the B-cell receptor pathway – the pathway in malignant B-cell growth and reproduction. In the US, there are about 5,000 new cases of MCL/year.

WM is a rare type of lymphoma that starts with a malignant change to the B-cell. There is no established standard of care or approved therapy. In the US, there are about 1,500 new cases each year.

Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis receives the 1st 2 US FDA Breakthrough Therapy Designations in FDA’s history (Reference Blog Post).


Pharmacyclics Clinical Trials Overview

Pharmacyclics Pipeline.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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