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FDA Approval for New Precision Medicine

Published: 2018-07-20 |

Source: Leukemia & Lymphoma Society

Progress in treating acute myeloid leukemia (AML), a blood cancer that takes more than 10,000 lives in the United States each year, took an important step forward today with the U.S. Food Drug Administration (FDA) approval of ivosidenib (Tibsovo) for patients who have specific genetic mutation called IDH1.

The Leukemia & Lymphoma Society (LLS), a leader in the offensive against AML, applauds today's FDA decision which provides a critical new option for patients with this particular subtype. Ivosidenib, developed by Agios Pharmaceuticals, is an oral, targeted therapy indicated for AML patients who have relapsed or do not respond to standard chemotherapy and have the IDH1 genetic marker, which is found in approximately 6 to 10 percent of the 20,000 people in the U.S. diagnosed with AML each year.

After a 40 year drought in the approval of new therapies for this lethal disease, today's approval marks the sixth approval for an AML drug in the past two years. Three of these approvals, including ivosidenib and its sister IDH2 inhibitor, enasidenib, are first-time approvals; the others are for new formulations, new indications, or reinstatement of agents approved previously. Through its research investment, LLS has played a significant role in helping to advance all of these approved therapies.

For the past four decades, patients younger than 60 years old with AML have been treated with the same two chemotherapy drugs followed in some cases with a blood stem cell transplant. These approaches are only partially effective. Moreover, most AML patients are older and cannot tolerate this therapy and the prognosis for these older AML patients is very poor, with less than 20 percent of these patients surviving five years after diagnosis.

Recent advances in molecular technology have enabled researchers to identify the underlying mutations in genes that can contribute to an individual patient's disease, opening the door to a more personalized approach to treatment. IDH1 and IDH2 are examples of these mutated genes.

"For so long, AML patients have desperately needed new and better options for treatment," said Louis J. DeGennaro, LLS President and CEO. "And for too long, AML has been treated as a one-size-fits-all disease, so it is encouraging to see these new treatments added to the armamentarium for AML patients. Today's approval is further proof that we are headed in the right direction with a precision medicine approach to conquering this difficult cancer."

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