Gaithersburg, Maryland- November 2012. A new potential treatment for a devastating rare seizure disorder has been granted “Orphan Drug” status by the Office of Orphan Products Development of the Food and Drug Administration (FDA). The designation makes Cerium eligible to receive certain market exclusivity, tax credits and fee waivers, if the medicine is approved in the United States for the designated indication.
Tetracosactide hexaacetate is a long acting synthetic adrenocorticotropic hormone (ACTH), which is used outside the United States in the treatment of a wide variety of diseases. The orphan drug designation was granted for the treatment of infantile spasms, which occurs in early infancy.
Infantile spasms, also know as West Syndrome, is an extremely rare condition affecting approximately 800 to 1800 babies per year in the U.S. Most cases of infantile spasms take place during the first year of life with the maximum incidence between 3 and 7 months. A unique seizure type characterizes the disorder and a substantial body of research indicates that the complete elimination of spasms should be the primary goal of treatment. Early diagnosis and prompt treatment are associated with positive long-term outcomes.
Available treatments in the U.S. include H.P. Acthar® Gel (animal based ACTH) and Sabril® (vigabatrin). While not currently approved in the U.S., long-acting tetracosactide is widely used in the treatment of infantile spasms around the world and is approved more than 50 countries.
Cerium Pharmaceuticals, Inc. is an emerging biopharmaceutical company dedicated to the development and commercialization of medicines for patients with rare (orphan) diseases. Cerium’s core focus is to acquire currently marketed products and mid-to-late stage development compounds for rare diseases with established project feasibility, leveraging basic discovery work already performed by others. Cerium’s unique business model pairs a core management team has extensive expertise in clinical development, regulatory affairs, and commercialization of rare disease medicines, linked to a network of academic researchers, specialized consultants, third party service providers and contractors. Cerium employs an optimized infrastructure to quickly develop and commercialize new orphan medicines.
About US Orphan Drug Designation
The US Orphan Drug Designation program provides orphan status to drugs intended to treat rare diseases or disorders that affect fewer than 200,000 people in the US, or that affect more than 200,000 people but are unlikely to recover the costs of developing and marketing the drug. Orphan designation by the FDA qualifies the sponsor for incentives provided for in the Orphan Drug Act, which can include design support for clinical trials, fee waivers, tax incentives, eligibility for expedited review, and seven years of market exclusivity upon approval.