The Breakthrough Therapy Designation is intended to expedite the development and review of a potential new drug for serious or life-threatening diseases where "preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development."[i] The designation of a drug as a Breakthrough Therapy was enacted as part of the 2012 Food and Drug Administration Safety and Innovation Act.
Pharmacylics, together with
"This is a historic moment in oncology. We are truly honored to have received this Breakthrough Designation and are pleased for patients and clinicians with the
The FDA Breakthrough Therapy Designation for ibrutinib in MCL patients was based on data from clinical and pre-clinical studies. Ibrutinib has the potential to improve the outcome in this serious and life-threatening disease which has a poor prognosis. The company and
- A Phase III randomized, multi-center registration trial of ibrutinib as a monotherapy versus temsirolimus in relapsed or refractory MCL patients who received at least one prior rituximab-containing chemotherapy regimen, RAY (MCL3001).
- A Phase III randomized, double-blind, placebo-controlled study of ibrutinib plus bendamustine and rituximab versus placebo plus bendamustine and rituximab in subjects with newly diagnosed MCL, SHINE (MCL3002).
- A Phase II single-arm study of ibrutinib as a monotherapy in patients with MCL who received at least one prior rituximab-containing chemotherapy regimen and who progressed after bortezomib therapy, SPARK (MCL2001).
- A Phase II single-arm study of ibrutinib as monotherapy in patients with relapsed or refractory MCL (PCYC-1104).
Further updates will be provided during the upcoming quarterly conference call on
About Mantle Cell Lymphoma
MCL is an aggressive type of B-cell non-Hodgkin lymphoma (NHL) that usually occurs in older adults. The disease typically begins in the lymph nodes but can spread to other tissues, such as bone marrow and liver. Ibrutinib targets the B-cell receptor pathway an important pathway in malignant B-cell growth and proliferation. In
About Waldenstrom's macroglobulinemia
Waldenstrom's macroglobulinemia is rare type of lymphoma. The disease begins with a malignant change to the B-cell during its maturation so that it continues to reproduce more malignant B-cells resulting in an overproduction of monoclonal immunoglobulin M antibody (IgM). WM is a hematologic malignancy for which no established standard of care - or approved therapeutic exists. In
Ibrutinib is an investigational drug designed to specifically target and selectively inhibit an enzyme called Bruton's tyrosine kinase (BTK). BTK is a key mediator of at least three critical B-cell pro-survival mechanisms occurring in parallel — regulating apoptosis, adhesion, and cell migration and homing. Through these multiple actions, BTK helps to direct malignant B-cells to lymphoid tissues, thus allowing access to a micro environment necessary for survival.
The effectiveness and safety of ibrutinib alone or in combination with other treatments is being studied in several B-cell malignancies, including chronic lymphocytic leukemia/small lymphocytic lymphoma, mantle cell lymphoma, diffuse large B-cell lymphoma, follicular lymphoma, Waldenstrom's macroglobulinemia and multiple myeloma. To date five Phase III trials have been initiated with ibrutinib and a total of 27 trials are currently registered on clinicaltrials.gov.
The Company is headquartered in
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