FDA approves rare blood cancer drug under breakthrough therapy designation

November 15, 2013

FDA has approved ibrutinib (Imbruvica, Pharmacyclics and Janssen) to treat patients with mantle cell lymphoma (MCL), a rare and aggressive type of blood cancer. It is the second drug with breakthrough therapy designation to receive FDA approval.

FDA has approved ibrutinib (Imbruvica, Pharmacyclics and Janssen) to treat patients with mantle cell lymphoma (MCL), a rare and aggressive type of blood cancer. It is the second drug with breakthrough therapy designation to receive FDA approval.

MCL, a rare form of non-Hodgkin lymphoma, represents about 6% of all non-Hodgkin lymphoma cases in the United States. By the time of diagnosis, MCL usually has affected the lymph nodes, bone marrow, and other organs.

Ibrutinib is indicated for patients with MCL who have received at least one prior therapy. It inhibits the enzyme needed by the cancer to multiply and spread.

Ibrutinib is the third drug approved by FDA to treat MCL. Bortezomib (Velcade, Millennium Pharmaceuticals) was approved in 2006 and lenalidomide (Revlimid, Celgene) was approved in 2013 to treat the disease.

“MCL is an orphan disease where a huge unmet need exists,” sai dMatt Outten, MBA, CPC, senior director of market access at Pharmacyclics. “Payers are looking for effective medications with high response rates. Payers will also appreciate the favorable risk-benefit profile of Ibrutinib.

“Imbruvica as a once-daily, oral therapy provides value for managed care and hospital decision-makers grounded in its overall clinical profile and mechanism of action,” said Mekré Senbetta, PharmD, Health Economics and Outcomes Research, Oncology, Janssen. “Managed care plans will now be able to offer their members an additional treatment option, and, it provides an important opportunity for payers to assess whether its efficacy and safety profile translates into cost savings to the health system.”

The drug’s accelerated approval for MCL is based on a study where 111 participants were given Ibrutinib daily until their disease progressed or side effects became intolerable. Results showed nearly 66% of participants had their cancer shrink or disappear after treatment. An improvement in survival or disease-related symptoms has not been established. 

FDA also granted ibrutinib priority review and orphan-product designation because the drug demonstrated the potential to be a significant improvement in safety or effectiveness in the treatment of a serious condition and is intended to treat a rare disease, respectively.

The most common side effects reported in participants receiving ibrutinib are thrombocytopenia, diarrhea, neutropenia, anemia, fatigue, musculoskeletal pain, edema, upper respiratory infection, nausea, bruising, dyspnea, constipation, rash, abdominal pain, vomiting, and decreased appetite. Other clinically significant side effects include bleeding, infections, kidney problems, and the development of other types of cancers.