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3 FDA Breakthrough Therapy Designations: Intercept Pharmaceuticals, Genentech, And Bluebird Bio

February 2, 2015

Three FDA Breakthrough Therapy Designations (BTDs) are announced in the past week by sponsor companies:

•   Intercept Pharmaceuticals on January 29th for Obeticholic Acid (OCA) for Nonalcoholic Steatohepatitis (NASH) with liver fibrosis

•   Genentech on February 2nd for cancer immunotherapy MPDL3280A (anti-PDL1), for PD-L1 (Programmed Death-Ligand 1) positive Non-Small Cell Lung Cancer (NSCLC)

•   Bluebird bio on February 2nd for its gene therapy product, LentiGlobin BB305, for transfusion-dependent patients with Beta-Thalassemia Major.

I – Intercept Pharmaceuticals And NASH

Intercept Pharmaceuticals, a New York-based biopharmaceutical company, is developing novel treatments for chronic liver diseases using its proprietary bile acid chemistry. On January 29th, the company announces the granting of the coveted FDA BTD to its lead investigational product candidate, Obeticholic Acid (OCA), for NASH with liver fibrosis.

OCA is being developed for 3 chronic liver diseases (reference Intercept Pharmaceuticals’ Pipeline):

•   NASH

•   Primary Biliary Cirrhosis (PBC)

•   Primary Sclerosing Cholangitis (PSC).

NASH is caused by the buildup of fat in the liver that results in liver damage and inflammation. NASH is part of a group of conditions called nonalcoholic fatty liver disease. Some interesting statistics about NASH:

•   3rd leading cause of liver transplants in the US

•   By 2020, NASH is expected to surpass Hepatitis C (HCV) as the leading cause of liver transplants.

OCA is a bile acid analog and 1st-in-class FXR (Farnesoid X Receptor) agonist. OCA has received Orphan Drug Designation (ODD) for the treatment of PBC and PSC in both the US and Europe. OCA also has FDA Fast Track Designation for PBC.

II – Genentech And NSCLC

Genentech, a member of the Roche Group, announces February 2nd that its investigational cancer immunotherapy, MPDL3280A, receives a 2nd FDA BTD. The 1st BTD is granted in May 2014 for Urothelial Bladder Cancer (UBC). Today’s BTD is for the treatment of patients with PD-L1 positive NSCLC, where the disease has progressed during or after platinum-based chemotherapy (and an appropriate targeted therapy for those with an EGFR mutation-positive or ALK-positive tumor).

Two FDA BTDs For MPDL3280A

Num Drug Name Indication
1 MPDL3280A (anti-PDL1) Urothelial Bladder Cancer (UBC)
2 MPDL3280A (anti-PDL1) Non-Small Cell Lung Cancer (NSCLC)


MPDL3280A (also known as anti-PDL1 and RG7446) is an investigational monoclonal antibody designed to interfere with a protein called PD-L1. MPDL3280A is designed to target PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, preventing it from binding to PD-1 and B7.1 on the surface of T cells. By inhibiting PD-L1, MPDL3280A may enable the activation of T cells, restoring their ability to effectively detect and attack tumor cells.

Clinical studies for additional tumor types are being initiated in 2015.

III – Bluebird bio And Gene Therapy

Bluebird bio, a Cambridge, Massachusetts-based start-up biotechnology company that is developing gene therapies for genetic and rare diseases, announces February 2nd that its investigational gene therapy, LentiGlobin BB305 Drug Product, receives the FDA BTD for the treatment of transfusion-dependent patients with Beta-Thalassemia Major.

“LentiGlobin BB305 Drug Product aims to treat beta-thalassemia major and severe sickle cell disease by inserting a functional human beta-globin gene into the patient’s own hematopoietic stem cells ex vivo and then transplanting those modified cells to the patient through infusion into the bloodstream, also know as autologous stem cell transplantation. LentiGlobin BB305 drug product is currently used in two Phase 1/2 studies for the treatment of beta-thalassemia: the Northstar Study (HGB-204) in the United States, Australia and Thailand, and HGB-205 in France.”  

Beta-Thalassemia is an inherited blood disorder caused by a genetic abnormality of the beta-globin gene resulting in defective red blood cells (RBCs). Patients with Beta-Thalassemia major (the severest form of beta-thalassemia) receive chronic blood transfusions, with the purpose of maintaining a steady state of hemoglobin levels for treating their severe anemia.

In November 2014, Spark Therapeutics receives a BTD for their gene therapy, SPK-RPE65, for the treatment of Nyctalopia (night blindness) for Leber’s congenital amaurosis.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Flickr – Nicholas T – Bows” by Nicholas A. Tonelli from Pennsylvania, USA (Bows) [CC-BY-2.0] | Wikimedia Commons.

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