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FDA Breakthrough Therapy Designation: First Drug Receives FDA Approval

November 1, 2013

The media, investors, patients, and the pharmaceutical industry are watching closely those drugs that receive the FDA Breakthrough Therapy Designation (BTD), to see what impact if any, the new coveted FDA Designation has on the amount of time it takes to receive FDA approval. The debate is ongoing, ever since the President’s signing of FDASIA (FDA Safety and Innovation Act), in July 2012.

The FDA announces on November 1, that Genentech’s orphan drug Gazyva (Obinutuzumab), receives FDA approval for use in combination with Chlorambucil (chemotherapy), to treat patients with previously untreated Chronic Lymphocytic Leukemia (CLL). The approval comes with a boxed warning regarding the Hepatitis B virus and a rare disorder that damages the material that covers and protects nerves in the white matter of the brain (Progressive Multifocal Leukoencephalopathy). The approval is a month and a half before the FDA PDUFA (Prescription Drug User Fee Act) Date of December 20, 2013.

It is the first drug with the FDA BTD to receive FDA approval. The following are the regulatory actions for Gazyva for the CLL indication:

•   FDA grants    Orphan Drug Designation                             February 2012
•   FDA grants    BTD #13                                                        May 2013
•   FDA accepts  Biologics License Application (BLA)             July 2013
•   FDA grants    Priority Review                                               July 2013
•   FDA grants    Approval                                                          November 1, 2013
•   FDA Scheduled PDUFA Date                                                December 20, 2013.

In the United States in 2013:

•   An estimated 15,600 patients will be diagnosed with CLL
•   An estimated 4,600 will die from CLL.

Gazyva will be available to patients in the United States within approximately two weeks. For those who qualify, Genentech plans to offer patient assistance programs for people taking Gazyva through Genentech Access Solutions.

A marketing application is submitted to the European Medicines Association (EMA) in April 2013. Also, in April 2013, another orphan drug, Pharmacyclics and Janssen’s Ibrutinib, receives the FDA BTD for CLL.

Time and additional approvals of BTD drugs will identify whether or not the BTD impacts the overall time to FDA approval. Until then, all stakeholders will continue to closely monitor, debate, and blog about the issue.

Please Note: FDA Official Logo from FDA website.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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