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Gilead Sciences Receives 19th FDA Breakthrough Therapy Designation

August 23, 2013

Gilead Sciences discloses during the company’s Q2 2013 Earnings call in July 2013, that the non-orphan Sofosbuvir + Ledipasvir oral Fixed Dose Combination (FDC) is granted the FDA Breakthrough Therapy Designation (BTD) for the treatment of Hepatitis C (HCV) :

“ … Following approximately one year behind the sofosbuvir single agent is the sofosbuvir/ledipasvir fixed dose combination which was granted breakthrough designation by the U.S. Food and Drug Administration.”

This is the 3rd FDA BTD for the treatment of Hepatitis C:

  1. AbbieVie’s 3-drug combination
  2. Bristol-Myers Squib (BMS)’s 3-drug regimen
  3. Gilead Sciences’ Sofosbuvir/Ledipasvir FDC.

Sofosbuvir is a second generation uridine nucleoside analogue which inhibits the NS5B protein of HCV. Ledipasvir is a direct-acting macrocyclic antiviral agent and an inhibitor of NS5A serine protease, an enzyme that is essential for viral replication.

Sustained Virologic Response (SVR) is the goal of HCV treatment. Interferon and Ribavirin, the conventional treatment for HCV, doesn’t eliminate the HCV virus from one’s liver, but it can suppress the virus to undetectable levels for an extended period of time. This is called a “sustained virologic response” or sustained response. It signifies that during the 6 months after one completes treatment, there is no detectable HCV in one’s blood. SVR is a good thing.

Impressive interim results are reported from Gilead Sciences’ Phase II study,  LONESTAR. In this study, the once-daily Sofosbuvir/Ledipasvir FDC is given with and without Ribavirin (RBV) to 60 treatment-naïve participants who don’t have cirrhosis. The reported SVR results are staggering:

  1. 19/19 or 100% of the participants in the 12 week-arm (without Ribavirin) experienced a SVR 4 weeks after completing therapy – an SVR 12 weeks after therapy is considered a cure
  2. 40/41 or 97.6% of participants undergoing the therapy for the 8-week arm (21 took ribavirin & 20 didn’t) experienced an SVR 8 weeks after stopping therapy.

There are two additional cohorts in the LONESTAR study evaluating a 12-week course of the Sofosbuvir/Ledipasvir FDC with or without Ribavirin in patients who previously fail therapy with an HCV-specific protease inhibitor-based regimen. Again, the SVR results are impressive:

  1. 95% of patients in each arm achieve a SVR 4 weeks after completing therapy.

The Blog Post, “FDA Breakthrough Therapy Designation Chart”, will always have the latest FDA Breakthrough Therapy (BT) designations, so please bookmark using URL

The latest “FDA Breakthrough Therapy Designation Statistics Chart” Blog Post is located at URL, so please bookmark.

Please Note: FDA Official Logo from FDA website.

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

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