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FDA Breakthrough Therapy Designation: Second Drug Receives FDA Approval

November 14, 2013

The FDA approves on November 13, the second drug to have the coveted Breakthrough Therapy Designation (BTD). The approval is for Pharmacyclics’ orphan drug Imbruvica (Ibrutinib) for the treatment of patients with Mantle Cell Lymphoma (MCL), a very rare and aggressive type of blood cancer.

The FDA’s BTD has come through once again, twice within the last 2 weeks.  November 1 saw the FDA’s first approval of a BTD for a drug – Genentech’s Gazyva (Obinutuzumab) for the treatment of patients with previously untreated Chronic Lymphocytic Leukemia (CLL).

The FDA approves Imbruvica under its accelerated approval program, which allows the agency to approve a drug to treat a serious or rare disease based on clinical data that shows that “the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients.”  Imbruvica also receives FDA’s Orphan Drug Designation (ODD) and Priority Review.

FDA Regulatory Actions For Imbruvica For MCL

•   FDA grants ODD December 2012

•   FDA grants BTD  February 2013

•   Pharmacyclics submits a FDA New Drug Application (NDA)  for Ibrutinib for 2 indications in July 2013 :
1.   Mantle Cell Lymphoma (MCL)
2.   Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL)

•   FDA accepts the filing of the July 2013 NDA for MCL & CLL/SLL  & Priority Review is granted August 2013

•   FDA approval of Imbruvica for MCL November 13, 2013.

Ibrutinib’s 6 FDA ODDs

Row Num

Designation Date

Orphan   Designation


10-23-2013 Diffuse Large B-Cell Lymphoma


10-15-2013 Waldenstrom’s Macroglobulinemia


12-03-2012 Mantle Cell Lymphoma


04-06-2012 Chronic Lymphocytic Leukemia


05-30-2013 Small Lymphocytic Lymphoma


05-16-2013 Multiple Myeloma

Ibrutinib’s 3 FDA BTDs

Row Num

Drug Name



Ibrutinib Relapsed Or Refractory Mantle Cell Lymphoma (MCL)


Ibrutinib Waldenstrom’s   Macroglobulinemia


Ibrutinib Chronic or Small Lymphocytic Leukemia (CLL/SLL)

The Column in the Chart titled “Drug Name” contains the URL for the link to the Press Release announcing the FDA Breakthrough Therapy Designation for each drug.

Please Note: “DNA Purification” Courtesy of Dr. Bruce Chassey Laboratory. National Institute Of Dental Research. Li-shan. NCI [Public domain in the US].

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


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