FDA OKs Additional Use for J&J Orphan Drug

The US Food and Drug Administration has approved use of Janssen Biotech’s and Pharmacyclics’ Imbruvica (ibrutinib) for patients with Waldenström's macroglobulinemia (WM), a rare form of cancer that begins in the body's immune system. The drug received a breakthrough therapy designation for this use. Janssen Biotech is one of the Janssen Pharmaceutical Companies of Johnson & Johnson.

A type of non-Hodgkin lymphoma, WM usually gets worse slowly over time and causes abnormal blood cells, known as B lymphocytes (B-cells), to grow within the bone marrow, lymph nodes, liver, and spleen. In WM, abnormal B-cells also overproduce a protein known as immunoglobulin M or IgM (macroglobulin) that may lead to excess bleeding, problems with vision and with the nervous system.

The FDA initially granted Imbruvica accelerated approval in November 2013 for use in patients with mantle cell lymphoma who received one prior therapy. In February 2014, the FDA granted accelerated approval to Imbruvica for use in patients with previously treated chronic lymphocytic leukemia (CLL), and then in July 2014, expanded its use to include treatment of CLL patients who carry a deletion in chromosome 17.

The FDA granted Imbruvica for WM breakthrough therapy designation, priority review, and orphan product designation because the company demonstrated through preliminary clinical evidence that the drug may offer a substantial improvement over available therapies; has potential, at the time of the application was submitted, to be a significant improvement in safety or effectiveness in the treatment of a serious condition; and the drug is intended to treat a rare disease, respectively. The product's new use is being approved more than two months ahead of its prescription drug user fee goal date of April 17, 2015, the date the FDA was scheduled to complete review of the drug application.

Source: FDA

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