Rare Disease Report

FDA Approves Adcetris for Rare Skin Cancers

NOVEMBER 09, 2017
James Radke, PhD
The U.S. Food and Drug Administration (FDA) has approved Adcetris (brentuximab vedotin) to treat 2 rare skin cancer: primary cutaneous anaplastic large cell lymphoma (pcALCL) and CD30-expressing mycosis fungoides (MF), both of which are common subtypes of cutaneous T-cell lymphoma (CTCL).

CTCL accounts for about 4% of non-Hodgkin lymphoma (i.e. approximately 2,800 patients in the U.S.) in which malignant lymphocytes affect the skin. pcALCL is characterized by its anaplastic cytology and the expression of the CD30 antigen. Typical symptoms of MF can include rash, tumors, skin lesions, and itchy skin.
Adcetris is an antibody directed therapy that targets CD30, the protein found on many pcALCL cells and on a proportion of MF cells.

The approval of Adectris for pcALCL and CD30-postive MF was largely based on a phase 3 trial and 2 phase 2 trials.

In the phase 3 trial, 128 pcALCL and CD30-expressing MF patients requiring systemic therapy were given Adcetris or standard therapy (methotrexate or bexarotene) and the study met its primary endpoint [overall response rate at 4 months (ORR4)]. More specifically, the ORR4 in Adcetris-treated group was 56.3% compared to 12.5% in the control group (P < .001).

The most common adverse events (AEs) were anemia, peripheral sensory neuropathy, nausea, diarrhea, fatigue and neutropenia.

The new indication is the 4th approved by the FDA for Adcetris, and was announced more than a month in advance of its expected PDUFA data. The drug is also approved for:
  • classical Hodgkin lymphoma (cHL) patients who fail autologous hematopoietic stem cell transplantation (auto-HSCT) or who fail at least two prior multi-agent chemotherapy regimens and are not auto-HSCT candidates,
  • patients with cHL at high risk of relapse or progression as post-auto-HSCT consolidation
  • systemic anaplastic large cell lymphoma (sALCL) patients who fail at least one prior multi-agent chemotherapy regimen.

In a news release, Susan Thornton, cutaneous lymphoma patient and chief executive officer of the Cutaneous Lymphoma Foundation (CLF) said,  “Cutaneous T-cell lymphoma is a blood cancer of the skin with no known cure and few new treatment options. It is a disfiguring disease in dire need of more effective and durable treatment options to help keep this debilitating and painful disease at bay.”

Thorton added, “As both a patient and representative of the cutaneous lymphoma community, we welcome the FDA approval of Adcetris as a new treatment option for the most common subtypes of cutaneous T-cell lymphoma in patients who require systemic therapy and we look forward to sharing this important milestone with patients and physicians.”

Adcetris comes with a boxed warning for progressive multifocal leukoencephalopathy (PML). JC (John Cunningham) virus infection resulting in PML and death can occur in Adcetris-treated patients.

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