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Inside Health Policy – Companies With 'Breakthrough' Products Say Pathway's Implications Are Unclear

Inside Health Policy – Companies With 'Breakthrough' Products Say Pathway's Implications Are Unclear

Two companies jointly developing a cancer drug that recently received FDA’s new breakthrough status said the implications of the designation are unclear and they are working with FDA to identify the pathway’s implications on development activities. The cancer therapy ibrutinib received two breakthrough designations to treat two types of lymphoma, representing the first oncology designations since the accelerated development pathway was created with enactment of the FDA Safety and Innovation Act in July.

Pharmacyclics, Inc. and Janssen Research & Development LLC announced Tuesday (Feb. 12) that the oncology drug they are co-developing was granted breakthrough therapy designations for the treatment of patients with relapsed or refractory mantle cell lymphoma (MCL) and patients with Waldenstrom’s macroglobulinemia (WM), a rare type of lymphoma for which no approved therapy exists. The two potential indications represent the third and fourth breakthrough designations granted.

The drug was designed to target and selectively inhibit a key enzyme involved in the survival of malignant B-cells, and the drug’s effectiveness and safety is being studied for several B-cell malignancies, the companies said. The companies said the designation for use in MCL patients was based on data from clinical and pre-clinical studies, and the drug firms have designed a development program for that indication. The companies said they expect to file an application to market ibrutinib for use in MCL patients before the end of 2013 and will continue discussions with FDA about filing for the WM indication.

Pharmacyclics and Janssen said they are working with FDA to determine the affect of the breakthrough designations on development and filing requirements for the use of ibrutinib in patients with MCL and WM. Pharmacyclics said it will provide regular updates as further information on implementing the requirements of the designation are developed by the agency.

The breakthrough pathway is intended to speed up the development and review of treatments for serious or life-threatening diseases and improve upon existing therapies. FDA officials said they intend to hold frequent meetings with companies developing drugs with this designation. The agency is still working out the details of how the new pathway will work, and plans to issue guidance on the breakthrough pathway and the other expedited pathways it employs.

“We are pleased that the FDA has granted two Breakthrough Therapy Designations for ibrutinib as the designation represents a major leap forward in accelerating drug development timelines,” said Paul Stoffels, chief scientific officer and worldwide chairman of pharmaceuticals at Johnson & Johnson, of which Janssen is a part. “We are committed to realizing the full potential of ibrutinib for patients with mantle cell lymphoma, Waldenstrom’s macroglobulinemia, as well as other B-cell malignancies, and will work with Pharmacyclics and the FDA to ensure the clinical development program for ibrutinib continues to move forward as quickly as possible.”

Ibrutinib is the first oncology drug to receive the breakthrough designation. Vertex Pharmaceuticals announced last month that it received FDA’s first two breakthrough drug designations for expanded uses of its cystic fibrosis drug Kalydeco. Vertex expressed similar sentiments, saying the implications of the new pathway are not yet determined.

Friends of Cancer Research, which helped develop the breakthrough pathway and secure its inclusion in FDASIA, said ibrutinib receiving the designation is “an example of progress and hope for patients fighting a range of cancers.” Stakeholders had said the accelerated pathway could be useful for targeted cancer drugs during debate around FDASIA.

“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,” Ellen Sigal, chair and founder of Friends of Cancer Research, said in a statement. “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.”