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Janssen Pharmaceutical Receives FDA Breakthrough Therapy Designation For Multiple Myeloma

May 6, 2013

Janssen Research & Development announces May 1, 2013, that non-orphan drug Daratumumab receives FDA’s Breakthrough Therapy Designation. The designation is for the treatment of patients with Multiple Myeloma (MM), who have received at least three prior lines of therapy including a Proteasome Inhibitor (PI) and an Immunomodulatory agent (IMiD), or who are double refractory to a PI and IMiD.

Daratumumab is an investigational human monoclonal antibody (mAb) that targets the CD38 molecule, “which is highly expressed on the surface of MM cells and may also have potential in other cancers on which CD38 is expressed”. The drug is currently in Phase I/II trials for MM and has potential against other malignancies on which CD38 is expressed. Daratumumab is discovered by the Danish company Genmab A/S. In August 2012, Genmab A/S and Janssen Research & Development enter into an agreement, that gives Janssen Research & Development an exclusive worldwide license to develop and commercialize the drug.

Daratumumab’s FDA Breakthrough Therapy Designation is the second oncology compound within the Janssen Research & Development pipeline to receive this designation. In the last three months, Pharmacyclics and Janssen Research & Development’s cancer therapy drug Ibrutinib receives 3 FDA Breakthrough Designations for :

•   Relapsed or refractory Mantle Cell Lymphoma (MCL – orphan designation)
•   Waldenstrom’s Macroglobulinemia (WM)
•   Chronic Lymphocytic Leukemia (CLL – orphan designation) or Small Lymphocytic Lymphoma (SLL) with deletion of the short arm of chromosome 17.

Multiple Myeloma Facts

•   Is a cancer of plasma cells
•   Per the American Cancer Society 2013 US estimates, 22,350 new cases will be diagnosed & 10,710 deaths will occur
•   5-Year relative survival rate is approximately 40%
•   There is no currently available cure.

11 Breakthrough Therapy Designations

The 11 Breakthrough Therapy Designations granted for drugs that have been identified by the sponsor companies (as of 05/06/13) are as follows :

•   Two are awarded to Vertex Pharmaceuticals for expanding the uses of its Cystic Fibrosis orphan drug Kalydeco
•   Three are awarded to Pharmacyclics and Janssen Research & Development for the cancer therapy drug Ibrutinib for relapsed or refractory Mantle Cell Lymphoma (MCL – orphan designation), Waldenstrom’s Macroglobulinemia (WM), and Chronic Lymphocytic Leukemia (CLL – orphan designation) or Small Lymphocytic Lymphoma (SLL) with deletion of the short arm of chromosome 17
•   One is awarded to Novartis for cancer non-orphan drug LDK378 for ALK+ Non-Small Cell Lung Cancer
•   One is awarded to Pfizer for breast cancer non-orphan drug Palbociclib
•   One is awarded to Merck for cancer non-orphan drug Lambrolizumab for Melanoma
•   One is awarded to Bristol-Myers Squibb for experimental, non-orphan three-drug regimen for Hepatitis C
•   One is awarded to Scioderm for investigational non-orphan product SD-101 for Epidermolysis Bullosa (EB)
•   One is awarded to Genmab and Janssen Research & Development for non-orphan drug Daratumumab for Multiple Myeloma.

The question that investors and patients have been asking is whether or not the new FDA Breakthrough Therapy will accelerate the approval process for the drugs receiving this designation. According to a FierceBiotech.com article, Novartis may be the first company “to put the proposition to a test”. Per this article, Novartis’ development team says in discussions with Bloomberg, that the company “could file for an approval of the breakthrough drug LDK378 after Phase II, which is typically the time when developers prep a lengthy late-stage effort that can prove a new cancer drug can prolong lives – the overall survival gold standard …”. That would put a new drug application at the FDA in early 2014.

Please Note #1: The Orphan Druganaut Blog has decided to include a Post on Janssen Research & Development’s drug Daratumumab even though it isn’t an orphan drug since the Blog has been following the new FDA Breakthrough Therapy designation from the beginning of 2013.

Please Note #2: FDA Official Logo from FDA website.

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

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