TUESDAY, JUNE 19, 2018

Pharmacyclics announces BTD for investigational oncology drug

Pharmacyclics, a clinical-stage biopharmaceutical company, announced on Tuesday that the U.S. Food and Drug and Administration had granted a breakthrough therapy designation for an investigational drug to treat patients with relapsed or refractory mantle cell lymphoma.

The FDA also granted the designation to ibrutinib monotherapy for the treatment of patients with Waldenstrom's macroglobulinemia. MCL and WM are both B-cell malignancies.

The BTD is meant to facilitate the development and review of potential new drugs for serious or life-threatening diseases when preliminary clinical evidence indicates the drug may significantly improve upon existing therapies.

"This is a historic moment in oncology," Bob Duggan, the CEO and chairman of the board of Pharmacyclics, said. "We are truly honored to have received this breakthrough designation and are pleased for patients and clinicians with the FDA's decision to expedite the development of ibrutinib. This compound entered the clinic in 2009 and has demonstrated tremendous clinical progress over the past four years. I would like to thank our collaboration partner, Janssen, for their support. Together we are committed to bringing this new therapeutic to patients and health care providers."

Pharmacyclics is working with Janssen and the FDA to determine the implications of the designation to the ongoing and planning development and filing requirements for using ibrutinib in patients with WM and MCL.

"As an oncology product, ibrutinib receiving the BTD is an example of progress and hope for patients fighting a range of cancers," Ellen Sigal, the chair and founder of Friends of Cancer Research, said. "This designation shows that the FDA is dedicated to using an 'all hands on deck approach' to work on products that show promise in treating serious and life-threatening diseases. The breakthrough pathway that our organization worked to create is intended to speed up the development and review of treatments that may demonstrate substantial improvement over existing therapies, and ibrutinib is a great example of using this new designation to potentially accelerate patient access to promising treatments."

In Phase II and Phase III trials, ibrutinib was shown to have potential to improve the outcome in patients with MCL. MCL is an aggressive type of B-cell non-Hodgkin lymphoma with a poor prognosis.