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Pharmacyclics, J&J's Janssen Now Confirming FDA Approval of IMBRUVICA

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Pharmacyclics, Inc. (NASDAQ: PCYC) today announced that the U.S. Food and Drug Administration (FDA) has approved IMBRUVICA™ (ibrutinib) as a single agent for the treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.1 This indication is based on overall response rate (ORR). An improvement in survival or disease-related symptoms has not been established. IMBRUVICA is a new agent that inhibits the function of Bruton's tyrosine kinase (BTK).1 BTK is a key signaling molecule of the B-cell receptor signaling complex that plays an important role in the survival of malignant B cells.2,3,4 IMBRUVICA blocks signals that stimulate malignant B cells to grow and divide uncontrollably.1,5

The FDA granted IMBRUVICA Breakthrough Therapy Designation due to the ORR and duration of response (DOR) seen in the Phase II study, PCYC-1104, and the serious and life-threatening nature of MCL.

The approval was based on the results of a multi-center, international, single-arm trial of 111 patients with previously treated mantle cell lymphoma. Tumor response was assessed according to the revised International Working Group (IWG) for non-Hodgkin lymphoma (NHL) criteria. The efficacy results demonstrated a 65.8% overall response rate (95% CI: 56.2, 74.5); 17% of patients achieved a complete response and 49% of patients achieved a partial response. The median duration of response was 17.5 months (95% CI: 15.8, not reached).

Safety was evaluated in the same 111 patients. The most common Grade 3 or 4 non-hematological adverse reactions (greater than or equal to 5%) were: pneumonia (7%), abdominal pain (5%), atrial fibrillation, diarrhea (5%), fatigue (5%), and skin infections (5%). Five percent of patients had Grade 3 or higher bleeding events (subdural hematoma, gastrointestinal bleeding, and hematuria). Treatment-emergent Grade 3 or 4 cytopenias (adverse reactions and laboratory abnormalities combined) were reported in 41% of patients. The Warnings and Precautions listed in the Prescribing Information include hemorrhage, infections, myelosuppression, renal toxicity, second primary malignancies and embryo-fetal toxicity.

Ten patients (9%) discontinued treatment due to adverse reactions in the trial.

Adverse reactions leading to dose reduction occurred in 14% of patients.

The recommended dose in this patient population is 560 mg (four 140 mg capsules) orally once daily.1

IMBRUVICA is one of the first treatments to receive FDA approval via the Breakthrough Therapy Designation pathway. Pharmacyclics completed its New Drug Application submission to the FDA in late June 2013 and received approval just over four months later.

"This is a meaningful day for previously treated mantle cell lymphoma patients, who are in need of new treatment options," said Michael Wang, M.D., Department of Lymphoma/Myeloma, The University of Texas MD Anderson Cancer Center and lead investigator for the pivotal registration trial PCYC-1104. "With IMBRUVICA, we now have an important new medicine that is a once-daily oral therapy with a favorable risk-benefit profile."

IMBRUVICA is commercially available immediately.

"The approval of IMBRUVICA marks significant progress toward Pharmacyclics' goal of serving as an ally to patients. After observing early signs of efficacy and tolerability of IMBRUVICA four years ago, we single-mindedly focused our attention on fully developing this medicine. We continue to explore IMBRUVICA's potential to treat cancer patients in need. Presently we are in the midst of investigating this medicine in numerous additional B-cell malignancies with 37 clinical studies ongoing," said Bob Duggan, CEO and Chairman of the Board of Pharmacyclics. "Today we celebrate the first approval of IMBRUVICA. I would like to thank the patients and physicians for their trust and participation in our clinical trials. We are very grateful to the FDA for their collaboration and support, and a very big 'thank you' to the entire Pharmacyclics & Janssen team who have tirelessly forwarded our mutual vision and mission to serve as allies for patients and physicians in the fight against cancer."

"Breakthrough Therapy Designation is intended to speed up the development and review of treatments to help address serious or life-threatening diseases. It is gratifying to see this early example of the new pathway meeting its intention – getting a promising treatment to patients who are waiting for new options," said Dr. Ellen Sigal, chair and founder of Friends of Cancer Research, a think tank and advocacy organization based in Washington, DC.

Posted-In: News FDA


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