The U.S. Food and Drug Administration (FDA) has approved Poteligeo (mogamulizumab-kpkc), a targeted treatment for people with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS), who have previously tried at least one systemic therapy. This is the first drug indicated specifically for the treatment of Sézary syndrome. MF and SS are the two most common types of cutaneous T-cell lymphoma (CTCL).
Refractory conditions are those that have not responded to previous treatment. Poteligeo is administered by injection for intravenous infusion. It is a monoclonal antibody that binds to a protein in some cancer cells.
Approval based on clinical trial data
FDA approval was based on a clinical trial of 372 adults with relapsed or refractory MF or SS. Trial participants were either given Poteligeo or the chemotherapy agent vorinostat. The trial measured the survival time of participants. Progression-free survival is defined as the amount of time someone stays alive without the cancer growing. Progression-free survival was more than double for patients taking Poteligeo compared to those receiving vorinostat. The median was 7.6 months compared to 3.1 months.1
How Poteligeo works
Poteligeo is a monoclonal antibody that binds to a protein in some cancer cells. Poteligeo targets the protein CC chemokine receptor type 4 (CCR4), which is found at high levels in leukemia and certain lymphoma subtypes, such as CTCL. When it binds to CCR4, Poteligeo activates immune cells to attack the cancer cells containing the protein.
Poteligeo is manufactured by Kyowa Kirin, Inc. It is the first biologic agent targeting CCR4 to be available for patients in the U.S. It is already approved for use in Japan. The FDA approval process used Priority Review and considered the drug for Breakthrough designation to expedite the evaluation, such that if approved it could treat serious conditions, and offer demonstrated improvement in treatment safety or effectiveness. Poteligeo is also classified as an Orphan Drug, one that treats rare diseases.
Mycosis fungoides and Sézary syndrome are rare, difficult to treat, forms of non-Hodgkin lymphoma. Non-Hodgkin lymphoma is a blood cancer that starts in the lymphocytes, white blood cells, which are part of the immune system. In MF and SS, lymphocytes become cancerous and affect the skin. MF represents about 50% of lymphomas that begin in the skin. It results in itchy red rashes and skin lesions. MF can spread to other parts of the body. SS is a rare form of skin lymphoma that affects the blood and lymph nodes.1 Both can be disfiguring, and debilitating.2
Possible side effects of Poteligeo
According to the FDA, the most common side effects of Poteligeo are rash, infusion-related reactions, fatigue, diarrhea, musculoskeletal pain and upper respiratory tract infection. This is not a complete list of all possible side effects of Poteligeo. As when starting any new drug, you should discuss with your doctor all medications, supplements and vitamins you take.
Serious warnings of treatment with Poteligeo include the risk of dermatologic toxicity, infusion reactions, infections, autoimmune problems, and complications from stem cell transplants that use donor stem cells (allogeneic) after treatment with the drug.1
Important things to know about Poteligeo
Poteligeo is expected to be available to consumers before the end of 2018. The course of treatment is administered over a 28-day cycle by IV infusions. There are presently no warnings or associated risks of major birth defects or miscarriage while using Poteligeo during pregnancy. Yet, effective contraception is recommended while being treated with Poteligeo and continued for at least 3 months following the last dose.3
FDA approves treatment for two rare types of non-Hodgkin lymphoma. U.S. Food & Drug Administration. Available at https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/UCM616176.htm?utm_campaign=08082018_PR_FDA%20approves%20new%20drug%20for%20two%20rare%20types%20of%20non-Hodgkin%20lymphoma&utm_medium=email&utm_source=Eloqua. Accessed online August 9, 2018.
Kyowa Kirin Announces FDA Approval of Poteligeo® (mogamulizumab-kpkc) for the Treatment of Mycosis Fungoides and Sézary Syndrome.
Press Release PR Newswire August 8, 2018. Available at https://markets.businessinsider.com/news/stocks/kyowa-kirin-announces-fda-approval-of-poteligeo-mogamulizumab-kpkc-for-the-treatment-of-mycosis-fungoides-and-s%C3%A9zary-syndrome-1027443628. Accessed online August 9, 2018.
Poteligeo prescribing information. Available at https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/761051s000lbl.pdf. Accessed online August 9, 2018.