Genmab has announced that HuMax-CD4 for the treatment of Mycosis Fungoides (MF), which constitutes 75% of all Cutaneous T-cell Lymphomas (CTCL), has been designated an orphan drug by the US Food and Drug Administration (FDA). The US prevalence of MF is estimated at 16,000 to 20,000.
Mycosis fungoides (MF) is the most common type of cutaneous T-cell lymphoma. It is a low-grade lymphoma which primarily affects the skin. Generally it has a slow course and often remains confined to the skin. Over time, in about 10% of the cases, it can progress to the lymph nodes and internal organs.
Under the Orphan Drug Act, orphan drug designation gives companies access to protocol assistance to maximize the chance of success at the time of marketing authorization. Once approved by the FDA, an orphan drug is granted 7 years of market exclusivity during which directly competitive similar products cannot normally be placed on the market.
The FDA may grant orphan drug designation to products that treat a rare disease or condition which affects less than 200,000 people in the US. A drug may also receive orphan drug designation if it is used to treat a disease that affects more than 200,000 people in the US, but there is no reasonable expectation that the cost of developing and making available a drug for such disease or condition will be recovered from US sales.
“HuMax-CD4 has now been granted orphan drug status in both the United States and Europe,” said Lisa N. Drakeman, Ph.D., Chief Executive Officer of Genmab. “This designation highlights the importance of HuMax-CD4 as a potential new treatment for CTCL.”
HuMax-CD4 is a high affinity human antibody that targets the CD4 receptor on T-lymphocytes. Genmab has run two Phase II studies using HuMax-CD4 to treat CTCL, one in early stage patients and the other for patients with advanced disease, both of which achieved positive results.
Planning for the pivotal study with MF patients is underway.