According to a story from Market Screener, the biopharmaceutical company Imbrium Therapeutics LP recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation for the company’s investigational therapeutic product tinostamustine. This designation was granted to the drug as a treatment for T-cell prolymphocytic leukemia, a rare form of blood cancer.
About T-Cell Prolymphocytic Leukemia
T-cell prolymphocytic leukemia is a rare form of T-cell leukemia that is characterized by its aggression and propensity to spread to other parts of the body, like the skin, liver, lymph nodes, spleen, and bone marrow. It is among the most rare types of leukemia, and comprises only about two percent of small cell leukemia cases in adult patients. The precise cause of T-cell prolymphocytic leukemia is unclear. Certain genetic mutations may be risk factors. Patients often present with systemic illness at diagnosis, and symptoms can include skin changes and enlargement of affected organs, such as the liver, spleen, and lymph nodes. Known for its aggressive nature, T-cell prolymphocytic leukemia does not respond well to many treatments; chemotherapy is not effective in most cases. The introduction of the monoclonal antibody alemtuzumab has improved outcomes. Stem cell transplant is occasionally attempted. The five year survival rate is very low. To learn more about T-cell prolymphocytic leukemia, click here.
About Orphan Drug Designation
Orphan Drug Designation is typically reserved for therapies that are intended to treat diseases that regarded as rare. A rare disease is classified as any disease or condition that affects less than 200,000 people in the US. To qualify, the therapy must demonstrate advantages in effectiveness or safety over currently available treatments or must address a currently unmet medical need. This designation confers several benefits to the recipient company, such as the waiving of certain fees, tax breaks, and a period of market exclusivity lasting seven years if the drug ultimately gains approval for public use.
There is a serious need for new therapies for T-cell prolymphocytic leukemia, so the granting of this designation is a significant step forward in getting a new treatment to patients with this deadly and aggressive rare cancer. Tinostamustine is also in development as a treatment for a variety of other solid tumor cancers and blood cancers.
