miRagen Therapeutics announced that the Food & Drug Administration (FDA) has granted orphan drug designation to cobomarsen, for the treatment of T-cell lymphoma. Cobomarsen is an inhibitor of miR-155 currently being developed by miRagen in two clinical programs to address different types of T-cell lymphoma, including a Phase 2 trial for cutaneous T-cell lymphoma (CTCL) and a Phase 1 trial for adult T-cell leukemia/lymphoma (ATLL).

“This is an important milestone in the development of cobomarsen. We believe the FDA’s decision to grant cobomarsen orphan drug designation underscores the need for new treatments for T-cell lymphomas such as ATLL and CTCL,” said miRagen President and Chief Executive Officer William S. Marshall, Ph.D.  “In addition to the promising results we’ve observed for the potential treatment of patients with ATLL and CTCL, we believe that cobomarsen has the potential to be a broad-based therapy for the treatment of cancer patients with elevated levels of miR-155.”

T-cell lymphomas are lymphomas that affect T lymphocytes such as ATLL and CTCL, amongst others. Together they make up less than 15% of non-Hodgkin lymphomas in the United States. There are many types of T-cell lymphoma, but they are all fairly rare. Overexpression of microRNA-155 has been associated with poor prognosis in a variety of T-cell lymphomas and other blood cancers.

Orphan drug designation is granted by the FDA to drugs or biologics that demonstrate potential for diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States.  The designation can provide development and commercial incentives for designated compounds and medicines, including eligibility for seven years of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and exemption from FDA user fees.