Regulatory Focus™ > News Articles > In Landmark Move, First Breakthrough Product Obtains FDA Approval

In Landmark Move, First Breakthrough Product Obtains FDA Approval

Posted 01 November 2013 | By Alexander Gaffney, RAC

The US Food and Drug Administration (FDA) has announced its first-ever approval under the breakthrough product designation, a new approval pathway of sorts intended to expedite the process by which highly promising products reach the market.


Under the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, FDA was given the authority to use a new expedited approval program known as breakthrough product designation. As defined by FDA, breakthrough products are those intended to treat a serious condition based on preliminary clinical evidence that indicates the drug may demonstrate substantial improvement on clinically significant endpoints over available therapies.

Unlike the other designations or pathways (fast-track designation, priority review designation, or accelerated approval), breakthrough product designation isn't meant to expedite development based on shortened timelines or surrogate markers, but rather grant access to enhanced review tools such as all of the features of the fast track designation, as well as "intensive guidance" from FDA regarding the development of the product.

In June 2013, FDA released a new guidance document, Expedited Programs for Serious Conditions - Drugs and Biologics, intended to establish its expectations for the program and illustrate how sponsors could obtain the designation.

The guidance, however, came months after the first breakthrough product application was accepted by FDA. In January 2013, FDA accepted two applications from Vertex Pharmaceuticals, one for an expanded indication for Kalydeco (ivacaftor) (already approved to treat a specific genetic variant of cystic fibrosis) and the second a combination of Kalydeco and an experimental compound (VX-809).

First Breakthrough Approval

But despite its first-in status, Vertex will not have the distinction of being the first breakthrough product to be approved by FDA.

That distinction belongs to Roche's Gazyva (obinutuzumab), FDA announced on 1 November 2013.

The drug-submitted under a biologic license application (BLA) in July 2013 and given priority review status-has now been approved for the treatment of chronic lymphocytic leukemia (CLL) in combination with chlorambucil. Approximately 16,000 Americans are diagnosed with CLL each year, and nearly 5,000 die from the disease on an annual basis.

"This approval reflects the promise of the Breakthrough Therapy Designation program, allowing us to work collaboratively with companies to expedite the development, review and availability of important new drugs," said Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.

Because of the drug's small intended patient population-under 200,000 in the US-the drug has also been given orphan drug status, FDA said.

FDA Announcement

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