Gil Roth10.08.13
Cornerstone Pharmaceuticals has received Orphan Drug Designation from the FDA for CPI-613, the company's lead Altered Energy Metabolism Directed (AEMD) drug candidate, for the treatment of myelodysplastic syndrome (MDS).
MDS is a form of cancer that occurs when the blood-forming cells in the bone marrow are damaged, which leads to low numbers of one or more type of blood cells. In about one-third of MDS cases, the disease progresses to a rapidly growing cancer of bone marrow cells called acute myeloid leukemia (AML). Currently no cure for MDS exists for patients with this life-threatening disease.
Approximately 15,000 people are diagnosed with MDS in the U.S. each year. Cornerstone is testing CPI-613 as a monotherapy in the treatment of relapsed / refractory MDS patients in a recently launched Phase II MDS trial; it may also try to establish CPI-613 as an alternative treatment option for this disease.
Orphan drug designation is granted by the FDA to therapeutics intended to treat rare diseases that affect fewer than 200,000 individuals in the U.S.. The designation entitles Cornerstone Pharmaceuticals to seven years of U.S. marketing exclusivity for CPI-613 upon regulatory approval, as well as an opportunity to apply for grant funding from the U.S. government to defray costs of clinical trial expenses, tax credits for clinical research expenses, and a potential waiver of the FDA's application user fee.
In addition to MDS, Cornerstone previously received orphan drug designation for the use of CPI-613 in the treatments of AML and pancreatic carcinoma.
MDS is a form of cancer that occurs when the blood-forming cells in the bone marrow are damaged, which leads to low numbers of one or more type of blood cells. In about one-third of MDS cases, the disease progresses to a rapidly growing cancer of bone marrow cells called acute myeloid leukemia (AML). Currently no cure for MDS exists for patients with this life-threatening disease.
Approximately 15,000 people are diagnosed with MDS in the U.S. each year. Cornerstone is testing CPI-613 as a monotherapy in the treatment of relapsed / refractory MDS patients in a recently launched Phase II MDS trial; it may also try to establish CPI-613 as an alternative treatment option for this disease.
Orphan drug designation is granted by the FDA to therapeutics intended to treat rare diseases that affect fewer than 200,000 individuals in the U.S.. The designation entitles Cornerstone Pharmaceuticals to seven years of U.S. marketing exclusivity for CPI-613 upon regulatory approval, as well as an opportunity to apply for grant funding from the U.S. government to defray costs of clinical trial expenses, tax credits for clinical research expenses, and a potential waiver of the FDA's application user fee.
In addition to MDS, Cornerstone previously received orphan drug designation for the use of CPI-613 in the treatments of AML and pancreatic carcinoma.