In the European Union (EU), Orphan designation, or Orphan Medicinal Product designation, is granted to drugs or biologics intending to treat, diagnose, or prevent a rare, chronically debilitating, or life-threatening condition. These conditions must not affect more than 5 in every 10,000 people. For a therapy to qualify, either no other product exists, or the drug/biologic significantly improves upon current standards-of-care. Incentives earned by the drug developer include protocol assistance, fee reductions, and market exclusivity following drug approval. According to a recent article in OncLive, the European Commission (EC) recently granted Orphan designation to duvelisib (Copiktra) for peripheral T-cell lymphoma (PTCL).
This designation hinged upon data from the Phase 2 PRIMO study. 101 adults with PTCL enrolled. The overall response rate was 49% — even when including patients who had been treated with at least three lines of treatment before the study — with a complete response rate of 34%.
Beyond this designation, duvelisib has been approved in the EU for the treatment of adults with either refractory follicular lymphoma or relapsed/refractory chronic lymphocytic leukemia. While it received similar Orphan Drug status in the United States, its approval for PTCL was withdrawn in late 2021. Within the last six months, the U.S. Food and Drug Administration (FDA) shared that duvelisib could heighten the mortality risk in people with leukemia or lymphoma. The therapy also increased the risk of skin rashes, intestinal and pulmonary inflammation, diarrhea, and infections. As such, the FDA did not feel like its benefits outweighed its risks.
What is Peripheral T-cell Lymphoma (PTCL)?
Peripheral T-cell lymphomas are a group of aggressive and uncommon non-Hodgkin’s lymphomas that form from mature white blood cells called natural killer (NK) cells and T-cells. Its name refers to its location in the body, with “peripheral” being lymphoid tissues (spleen, skin, lymph nodes) beyond the bone marrow. This cancer is more common in men and people over 60 years old. Symptoms can, but do not always, include:
- Unintended weight loss
- Swollen but painless lymph nodes in the neck, groin, or armpits
- Night sweats
- Itchy rash
- Chest or abdominal pain
Treatment options include chemotherapy, stem cell transplantation, belinostat, romidepsin, pralatrexate, and investigational therapeutics.