Duvelisib Wins Orphan Drug Designation for PTCL in Europe


The European Commission has issued an orphan drug designation to duvelisib for the treatment of adult patients with peripheral T-cell lymphoma.

David Sidransky, MD

David Sidransky, MD

The European Commission (EC) has issued an orphan drug designation to duvelisib (Copiktra) for the treatment of adult patients with peripheral T-cell lymphoma (PTCL).1

The European Medicines Agency previously approved the oral PI3K inhibitor for the treatment of adults with relapsed/refractory chronic lymphocytic leukemia (CLL) or refractory follicular lymphoma (FL) in March 2021.2

“This orphan drug designation recognizes the significant unmet need in patients with PTCL, especially those with relapsed/refractory disease,” David Sidransky, MD, clinical advisor to Secura Bio and director of head and neck cancer research in the Department of Otolaryngology-Head and Neck Surgery and a professor of oncology at Johns Hopkins University, stated in a news release. “This patient group has very limited therapeutic options and duvelisib may offer a new choice of therapy.”

Investigators published findings from 101 patients with PTCL treated with duvelisib in the phase 2 PRIMO trial (NCT03372057) in November 2022. At a median follow-up of 8.7 months following first response, the overall response rate (ORR) was 49% with a complete response (CR) of 34%, according to an independent review committee. The ORR remained 49% even in patients who received at least 3 prior treatments. The median duration of response was 7.7 months; 7.4 months in those who had CR.3

The median patient age was 67.0 years (range, 21-92), and patients received a median of 3 prior lines of therapy (range, 1-9). Prior treatments included CHOP/R-CHOP, CHOEP/EPOCH, or BV/BV-chemo (36.6% each), salvage chemo after CHOP/R-CHOP or CHOEP/EPOCH (37.6%), and autologous stem cell transplant (21.8%). The mean treatment duration was 17.1 weeks, and the median treatment duration was 9.0 weeks (range, 1-79).

The FDA granted duvelisib an orphan drug designation for relapsed/refractory PTCL in 2019.4 The agency approved duvelisib for the treatment of patients with relapsed/refractory CLL and SLL or relapsed/refractory FL based on findings from the DUO trial and the phase 2 DYNAMO trial (NCT01882803) in 2018.5 However, in December 2021, Secura Bio withdrew the indication in the United States.6

The FDA warned in July 2022 that the agent may increase the risk for death in patients with leukemia and lymphoma compared with another agent. Duvelisib was also found to be associated with a higher risk for serious adverse effects such as infections, diarrhea, inflammation of the intestines and lungs, skin reactions, and high liver enzyme levels in the blood.7

In September 2022, the FDA’s Oncologic Drugs Advisory Committee determined that final overall survival (OS) data did not suggest a benefit-risk ratio strong enough to warrant support for the use of duvelisib in adults with relapsed/refractory CLL and SLL following at least 2 prior therapies. The agency cited safety concerns and findings showing that duvelisib was associated with a higher rate of deaths compared with ofatumumab (Kesimpta).8


  1. Secura Bio, Inc. receives orphan drug designation in Europe for duvelisib for the treatment of patients with peripheral T-cell lymphoma. News release. Secura Bio. January 9, 2023. Accessed January 10, 2023. https://yhoo.it/3k38klh
  2. Verastem Oncology receives orphan drug designation from FDA for COPIKTRA for the treatment of T-cell lymphoma. News release. Verastem Oncology. October 3, 2019. Accessed January 10, 2023. https://bwnews.pr/332o768
  3. Jacobsen ED, Zinzani PL, Zain J, et al. Duvelisib in patients with relapsed/refractory peripheral T-cell lymphoma from the phase 2 PRIMO trial expansion phase: impact of prior treatment and expanded safety analysis. Blood. 2022;140(suppl 1):9387-9389. doi:10.1182/blood-2022-169432
  4. Verastem Oncology receives FDA approval of COPIKTRA™ (duvelisib) Capsules. Published September 24, 2018. Accessed January 10, 2023. https://bit.ly/2NDQm80
  5. Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 March 2021. March 26, 2021. Accessed January 10, 2023. https://bit.ly/3ivytZK
  6. Secura Bio announces Copiktra (duvelisib) strategic focus on T-cell lymphoma and voluntary US withdrawal of the relapsed or refractory follicular lymphoma indication. News release. Secura Bio, Inc.; December 3, 2021. Accessed January 10, 2023. https://prn.to/31zvqH8
  7. FDA warns about possible increased risk of death and serious side effects with cancer drug Copiktra (duvelisib). News release. FDA. June 30, 2022. Accessed January 10, 2023. https://bit.ly/3y8ZJkp
  8. Oncologic Drugs Advisory Committee (ODAC) Meeting. FDA. September 23, 2022. Accessed January 10, 2023. https://www.youtube.com/watch?v=cjkgIpaxHlI
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