Rare Disease Report

Sickle Cell Drug Gets Fast Tracked

OCTOBER 03, 2014
RDR Staff

The U.S. Food and Drug Administration has granted Fast Track designation to NKTT120 for the treatment of sickle cell disease. 

NKTT120 is a humanized monoclonal antibody that depletes specific regulatory T cells (iNKT cells) that preclinical models indicate may be a key mediator of organ damage in sickle cell disease.
The FDA previously granted Orphan Drug designation to NKTT120 for the treatment of sickle cell disease. The added Fast Track designation is intended to facilitate development and expedite review. 

In a press release, Robert Mashal, chief executive officer of NKT Therapeutics, the developers of the drug,  said:

"We look forward to working with the FDA on the design of our efficacy trials for NKTT120."

"Fast Track status will help us reach our goal of bringing this new therapy to patients as rapidly as possible."

NKT Therapeutics also announced they have completed dosing in a Phase 1b trial of NKTT120 in patients with sickle cell disease.  The primary goal of the trial is to determine the safety of NKTT120 in such patients.  Preliminary results from this study will likely be presented later this year.  

About Sickle Cell Disease

Sickle cell disease is a rare genetic condition that affects 90,000 to 100,000 Americans, mostly African Americans.  A person with sickle cell disease has red blood cells that are hard, sticky, and C-shaped (like the farm tool the "sickle"). These cells clog smaller blood vessels resulting in pain as well as increased risk for infection, acute chest syndrome and stroke.

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