FDA Grants Breakthrough Therapy Designation for Pevonedistat to Treat Higher-Risk MDS

Takeda Pharmaceutical Company Limited announced the FDA granted pevonedistat, its investigation NEDD8-activating enzyme inhibitor, breakthrough therapy designation to treat patients with higher-risk myelodysplastic syndrome.

The FDA granted breakthrough therapy designation to pevonedistat for the treatment of patients with higher-risk myelodysplastic syndromes (HR-MDS), according to a press release from Takeda Pharmaceutical Company Limited.

The investigational drug, pevonedistat, is a NEDD8-activating enzyme (NAE) inhibitor that could be the first novel treatment for patients with HR-MDS in more than a decade.

“Higher-risk MDS is associated with poor prognosis, diminished quality of life and a higher chance of transformation to acute myeloid leukemia, another aggressive cancer,” Christopher Arendt, head of Oncology Therapeutic Area Unit for Takeda, said in a press release. “The combination of pevonedistat and azacitidine is a promising therapeutic approach with the potential to be the first novel treatment advancement for higher-risk MDS in more than 10 years.”

The breakthrough therapy designation comes in response to the final analysis of the Pevonedistat-2001 phase 2 study evaluating pevonedistat plus azacitidine (Vidaza) versus azacitidine alone to treat patients with HR-MDS among other rare leukemias.

Primary end points included overall survival, event-free survival, complete remission, and transfusion independence. The FDA also considered the adverse event profile of the results.

Takeda presented the initial results of the Pevonedistat-2001 phase 2 study at the virtual 56th American Society of Clinical Oncology (ASCO) Annual Meeting and virtual 25th European Hematology Association (EHA) Annual Congress.

Current treatment options for patients living with NR-MDS, including hypomethylating agent monotherapy, have resulted in poor outcomes.

The FDA grants breakthrough therapy designation to “accelerate the development and regulatory review of investigational drugs that are intended to treat serious or life-threatening ailments.” Drugs granted this designation have shown initial clinical evidence of substantial improvement over existing therapies.

MDS is a rare form of bone-marrow related cancer most commonly impacting patients with a median age of diagnosis from 60 to 74 years. The disease is caused by irregular blood cell production within the bone marrow.

Typical symptoms of MDS include fatigue, shortness of breath, easy bruising or bleeding, loss of appetite, weakness, pale skin, fever and frequent or severe infections.

“We thank the FDA for recognizing pevonedistat, and the urgency to develop innovative therapies that address critical treatment needs for higher-risk MDS, a patient population with few options,” Arendt said in a press release,


Takeda Announces U.S. FDA Breakthrough Therapy Designation Granted for Pevonedistat for the Treatment of Patients with Higher-Risk Myelodysplastic Syndromes (HR-MDS) [news release]. Cambridge, Massachusetts and Osaka, Japan. Published July 30, 2020. https://www.takeda.com/newsroom/newsreleases/2020/takeda-announces-u.s.-fda-breakthrough-therapy-designation-granted-for-pevonedistat-for-the-treatment-of-patients-with-higher-risk-myelodysplastic-syndromes-hr-mds/. Accessed July 30, 2020.

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