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FDA Breakthrough Therapy Designation: Third Drug Receives FDA Approval

December 8, 2013

The FDA approves on December 6, the third drug to have the coveted Breakthrough Therapy Designation (BTD). The approval is for Gilead Sciences’ non-orphan drug Sovaldi (Sofosbuvir) for the treatment of patients with chronic Hepatitis C (HCV). What is a “breakthrough” about this approval is the fact that Sovaldi is the first drug that safely and with efficacy, treats particular types of HCV without the need for co-administration of Interferon.

Per Gilead Sciences’ Press Release, approximately 4 million Americans are infected with HCV in the United States. The current standard of care for HCV is up to 48 weeks with a pegylated interferon (peg-IFN)/ribavirin (RBV)-containing regimen, depending on the patient’s particular HCV genotype. Sovaldi is a once-daily oral nucleotide analog polymerase inhibitor receiving approval for chronic HCV as a part of a combination antiviral treatment regimen. The drug blocks a protein used by HCV to replicate.

According to a Reuters online article, most patients will be treated with the $1,000-a-day drug for 12 weeks, resulting in a total list price of $84,000. Also, according to the same article, analysts, on average, have forecast Sovaldi sales of $1.9 billion for 2014, according to BMO Capital Markets.

Sovaldi Quick Facts

•   Receives FDA Priority Review

•   Shows high cure rates (SVR12) and shortened, 12-week course of therapy for many patients

•   Receives FDA approval for HCV Genotypes 1, 2, 3, & 4

•   1st ever oral treatment regimen for Genotypes 2 or 3

•   1st regimen for patients awaiting liver transplantation to prevent HCV recurrence.

Other Sovaldi Regulatory Actions

•   Receives on November 22, a positive opinion for an application for marketing authorization from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP)

•   Pending applications for marketing approval in Australia, Canada, New Zealand, Switzerland and Turkey.

FDA BTDs Receiving FDA Approval as of 12/06/13

•   11/01/13 – 1st  approval of a BTD – Genentech’s orphan drug Gazyva (Obinutuzumab) for previously untreated Chronic Lymphocytic Leukemia (CLL)

•   11/13/13 – 2nd approval of a BTD – Pharmacyclics’ orphan drug Imbruvica (Ibrutinib) for Mantle Cell Lymphoma (MCL)

•   12/06/13 – 3rd approval of a BTD – Gilead Sciences non-orphan drug Sovaldi (Sofosbuvir) for Hepatitis C.

Sovaldi is the second drug receiving approval by the FDA, in the past two weeks, to treat chronic HCV. On November 22, the FDA approves Olysio (Simeprevir) for chronic HCV. In 2011, the FDA approves Merck’s Victrelis (Boceprevir) and Vertex Pharmaceuticals’ Incivek (Telaprevir) for the treatment of hepatitis C.

The media, investors, patients, and the pharmaceutical industry have been 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. It will be interesting to see if the FDA approves additional BTD drugs by the end of 2013.

The latest “FDA CDER + CBER Breakthrough Therapy Designation Statistics Chart” will always be located at URL, so please bookmark.

The latest “FDA Breakthrough Therapy Designation Chart” Blog Post will always be located at URL, so please bookmark.

Please Note: “DNA Purification” Courtesy of Dr. Bruce Chassey Laboratory. National Institute Of Dental Research. Li-shan. NCI [Public domain in the US].

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


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