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FDA Approval of New Precision Medicine Therapy for Deadly Blood Cancer

Rye Brook, N.Y. (July 20, 2018) – 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 a 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.”

In 2016, LLS launched its own groundbreaking precision medicine study, the Beat AML Master Clinical Trial, a significant collaboration in which multiple targeted treatments for newly diagnosed AML patients are being tested simultaneously at multiple cancer centers around the country. While today’s approval of ivosidenib is for relapsed and refractory patients, the agent is being tested in the Beat AML Master Clinical Trial as a first-line therapy for newly diagnosed AML patients with the IDH1 mutation.

“The Beat AML Master Clinical Trial is designed to deliver the right drug to the right patient at the right time,” said DeGennaro. “As a patients-first organization, LLS is committed to leading and innovating new approaches to the way cancer research is conducted.”

To learn more about ivosidenib, click here.

More Promise for AML Treatment

LLS invests more than one-quarter of its research budget annually in AML research – more than $100 million over the past five years. In addition to the Beat AML Master Clinical Trial, LLS is currently supporting approximately 70 pre-clinical and clinical AML projects.

In fact, LLS is already investing in understanding the basis of resistance to IDH inhibitors to further improve outcomes for AML patients treated with IDH targeted therapies.

“LLS invests in research areas in which we know we can make a significant impact,” explains DeGennaro. “We recognized early on the urgency in finding new and better treatments for AML. After four decades and millions of dollars invested, we are finally seeing results for AML patients that give us a real reason for optimism.”

About The Leukemia & Lymphoma Society
The Leukemia & Lymphoma Society® (LLS) is the world's largest voluntary health agency dedicated to fighting blood cancer. The LLS mission: Cure leukemia, lymphoma, multiple myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care.

Founded in 1949 and headquartered in Rye Brook, NY, LLS has chapters throughout the United States and Canada. To learn more, visit www.LLS.org. Patients should contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET.

For additional information visit lls.org/lls-newsnetwork. Follow us on FacebookTwitter, and Instagram.

Contact:
Linda Heaney
Linda.Heaney@lls.org
914-821-8208