Regulatory Focus™ > News Articles > Pfizer Claims Fourth Breakthrough Product Designation, But Joins Others in Citing Unclear Benefit

Pfizer Claims Fourth Breakthrough Product Designation, But Joins Others in Citing Unclear Benefit

Posted 11 April 2013 | By Alexander Gaffney, RAC

Pharmaceutical manufacturing giant Pfizer has announced that its breast cancer candidate palbociclib has received a new and coveted breakthrough product designation from the US Food and Drug Administration (FDA), the third company and fourth product to announce their receipt of the designation to date.


Though many companies are seeking breakthrough product designation-40 requests in all through 31 March 2013, by FDA's count--It's not entirely clear what the designation will mean for companies in practical terms.

The designation was created under the 2012 FDA Safety and Innovation Act (FDASIA), and is intended to give special regulatory advantages to products intended to treat-often in combination with other products-patients with life-threatening or otherwise serious diseases for which there are no or few treatment options.

FDASIA defined eligibility for the pathway as a product that is "intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints."

The designation opens up new incentives for companies, giving their applications access to faster reviews. This is particularly important for combination therapies, which might not otherwise be eligible for priority review status or other incentives despite their intended use. The principal benefit, as explained by its advocates, is that novel combination therapies involving already-approved products will now be better able to obtain those faster review times, further assisting patients.

The review category has already seen 10 products accepted, but only two other companies-Massachusetts-based Vertex Pharmaceuticals and Johnson & Johnson subsidiary Janssen-have announced their status publicly.

Vertex, the first company to announce its breakthrough product designation status publicly, actually has two products set to go through the expedited pathway. The first, a combination of Kalydeco (ivacaftor) and an experimental compound known as VX-809, is intended to treat additional forms of cystic fibrosis. The second, just for Kalydeco, is also intended to treat additional variants of cystic fibrosis. At present, the drug is only approved to treat one variation of the disease which specifically afflicts patients with the G551D mutation.

Janssen, meanwhile, has received the designation for ibrutinib, an investigation oral Bruton's tyrosine kinase (BTK) inhibitor intended for use as a monotherapy with patients with chronic lymphocytic leukemia or small lymphocytic lymphoma with deletion of the short arm of chromose 17 (del17p)

Pfizer Makes four, but What Does it all Mean?

Pfizer's 10 April 2013 announcement now marks the fourth product publicly associated with the program.

But, as with Vertex and Janssen, Pfizer seemed to have little insight as to what practical effect the program would have on the drug's review schedule.

"Pfizer will continue to work with the FDA to better understand the implications of Breakthrough Therapy designation on the palbociclib development program and to generate evidence needed to support a potential regulatory submission. The FDA's requirements for a potential submission have not yet been defined," the company wrote in a statement.

"We appreciate the opportunity that Breakthrough Therapy designation provides to work closely with the FDA on the development of palbociclib," said Mace Rothenberg, senior vice president of clinical development and medical affairs for Pfizer's Oncology business unit.

Those statements comport closely with those of Vertex and Janssen.

"The implications of Breakthrough Therapy Designation cannot be determined at this time," Janssen wrote. "Janssen and Pharmacyclics are working with the FDA to determine any potential implications of the Breakthrough Therapy Designation to the ongoing and planned development activities."

"The implications of Breakthrough Therapy Designation cannot be determined at this time," Vertex wrote in a statement. "Vertex is working with FDA and other global regulatory agencies to determine any potential implications of the Breakthrough Therapy Designations to its ongoing and planned development activities, and subsequent regulatory submissions, for ivacaftor monotherapy and the combination regimen of VX-809 with ivacaftor."

More Information on the Way

The question, then, is whether the "opportunity" identified by all three companies will result in a meaningful benefit not otherwise available through various other pathways, including fast-track designation, accelerated approval, priority review or orphan drug designation.

More information could be coming soon. In a March 2013 meeting before a group of biotechnology executives, FDA Commissioner Margaret Hamburg said that a draft guidance document on breakthrough product designations would be available by year's end-time enough, perhaps, for currently designated companies to benefit.

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