FDA approves Poteligeo ( mogamulizumab-kpkc )

August 8, 2018, The U.S. Food and Drug Administration today approved Poteligeo (mogamulizumab-kpkc) injection for intravenous use for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy. This approval provides a new treatment option for patients with MF and is the first FDA approval of a drug specifically for SS.

“Mycosis fungoides and Sézary syndrome are rare, hard-to-treat types of non-Hodgkin lymphoma and this approval fills an unmet medical need for these patients,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “We are committed to continuing to expedite the development and review of this type of targeted therapy that offers meaningful treatments for patients.”

Non-Hodgkin lymphoma is a cancer that starts in white blood cells called lymphocytes, which are part of the body's immune system. MF and SS are types of non-Hodgkin lymphoma in which lymphocytes become cancerous and affect the skin. MF accounts for about half of all lymphomas arising from the skin. It causes itchy red rashes and skin lesions and can spread to other parts of the body. SS is a rare form of skin lymphoma that affects the blood and lymph nodes.

Poteligeo is a monoclonal antibody that binds to a protein (called CC chemokine receptor type 4 or CCR4) found on some cancer cells. 

The approval was based on a clinical trial of 372 patients with relapsed MF or SS who received either Poteligeo or a type of chemotherapy called vorinostat. Progression-free survival (the amount of time a patient stays alive without the cancer growing) was longer for patients taking Poteligeo (median 7.6 months) compared to patients taking vorinostat (median 3.1 months).

The most common side effects of treatment with Poteligeo included rash, infusion-related reactions, fatigue, diarrhea, musculoskeletal pain and upper respiratory tract infection.

Serious warnings of treatment with Poteligeo include the risk of dermatologic toxicity, infusion reactions, infections, autoimmune problems (a condition where the immune cells in the body attack other cells or organs in the body), and complications of stem cell transplantation that uses donor stem cells (allogeneic) after treatment with the drug.

The FDA granted this application Priority Review and Breakthrough Therapy designation. Poteligeo also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

POTELIGEO is used to treat adults with mycosis fungoides (MF) or Sézary syndrome (SS) who have received at least one prior systemic (such as injections or pills) treatment for their disease but the treatment did not work or the disease has come back.

MF and SS are rare types of cancer called cutaneous T-cell lymphoma which form in the lymph system and affect the skin.

How is this drug used?

POTELIGEO is given by a healthcare provider directly into the bloodstream through a needle in the vein. This is known as an intravenous, or IV infusion. It takes about one hour to receive a POTELIGEO infusion.

POTELIDEO is given on days 1, 8, 15, and 22 of the first 28-day cycle and on days 1 and 15 of each subsequent cycle.

What are the benefits of this drug?

In the trial, patients who received POTELIGEO experienced longer period of time a patient stays alive without the cancer growing (called progression-free survival) in comparison to patients who received vorinostat (a type of chemotherapy) . For patients treated with POTELIGEO it took about 7.6 months before disease progression and for patients treated with vorinostat about 3.1 months.

Were there any differences in how well the drug worked in clinical trials among sex, race and age?

• Sex: POTELIGEO worked similarly in men and women.
• Race: Most of the patients were White. Differences in how well the drug worked among races could not be determined because of the small number of patients in other races.
• Age: POTELIGEO worked similarly in patients younger and older than 65 years of age.