Orphan status for anti-CTLA-4 antibody
The FDA has granted orphan drug designation to Medarex's fully human anti-CTLA-4 antibody, MDX-010, for the treatment of high risk Stage II, Stage III and Stage IV melanoma.
The FDA has granted orphan drug designation to Medarex's fully human anti-CTLA-4 antibody, MDX-010, for the treatment of high risk Stage II, Stage III and Stage IV melanoma.
Orphan designation is granted for treatments that offer potential therapeutic value for diseases that affect fewer than 200,000 people in the US. Orphan Drug designation also provides eligibility for a special seven-year period of market exclusivity upon approval, potential tax credits for research, grant funding for research and development, reduced filing fees for marketing applications, and assistance with the review of clinical trial protocols.
Pending approval of a Special Protocol Assessment (SPA) application that has been filed with the FDA, Medarex expects to initiate a pivotal study for MDX-010 in combination with a gp100 melanoma vaccine in the second half of 2004. Positive data from a Phase II trial with MDX-010 alone and in combination with dacarbazine for the treatment of metastatic melanoma was recently presented at the annual meeting of the American Society of Clinical Oncology (ASCO). MDX-010 is also being tested in a Phase II chemotherapy combination clinical trial for prostate cancer and is being studied as a monotherapy in a Phase II clinical trial for breast cancer and a Phase I clinical trial for HIV.
'We are pleased with the orphan drug designation and believe that MDX-010 may have the potential to address the serious unmet medical needs faced by high risk melanoma patients,' said Donald Drakeman, president and ceo of Medarex.
About MDX-010
MDX-010 is a fully human antibody against human CTLA-4, a molecule on T cells that is believed to be responsible for suppressing the immune response. Data recently presented at ASCO from a Phase II study of MDX-010 in combination with dacarbazine showed a 17.1% response rate (6 of 35 patients) of complete and partial responses in patients with metastatic melanoma. In a separate Phase II study conducted last year, a 21.4% response rate (3 of 14) was observed in metastatic melanoma patients treated with a high 3mg/kg dose of MDX-010 in combination with gp100 peptide vaccine. In the MDX-010/vaccine combination clinical trial, patients who experienced complete and partial responses still remain cancer-free for over twenty months. Diarrhea and rash have been the most common Grade III or IV adverse events reported, and development of these events may suggest that breaking immune system tolerance may be associated with the development of durable complete and partial responses. According to the American Cancer Society, cancer of the skin is the most common of all cancers. Melanoma accounts for about 4% of skin cancer cases, but it causes about 79% of skin cancer deaths. The American Cancer Society estimates that in 2004 there will be about 55,100 new cases of melanoma in the United States, and about 7,910 people will die of this disease.