Sickle Cell Disease (SCD) is a group of genetic disorders that result in dysfunctional hemoglobin and a depletion of Cervonic Acid Esters in the walls of blood cells. These abnormalities increase the red and white blood cells’ tendency to adhere to each other, which results in episodic occlusions of blood vessels, reperfusion damage and excruciating pain. Ultimately, many children develop end organ damage and strokes. There are approximately 100,000 cases of SCD in the United States, which is estimated to represent a >$200MM market opportunity. The international demand for such a product is far greater than that of the United States. Treatment options are limited and associated with high risk of severe side effects.
Problem: There are no FDA approved therapies for children with Sickle Cell Disease, an Orphan Drug Candidate.
Current Treatment: Opioid pain medications, non-steroidal anti-inflammatory drugs (NSAIDS), off label hydroxyurea, transfusions and bone marrow transplants.
Orphan Drug Designation
Sancilio & Company, Inc. submitted an application to the Federal Drug Administration (FDA) requesting Orphan Drug Designation for SC411 for the treatment of Sickle Cell Disease. The designation was granted in April 2015. This designation may provide seven years of market exclusivity in the United States, subject to certain limited exceptions. However, the orphan drug designation does not convey any advantage in, or shorten the duration of, the regulatory review or approval process.
Orphan Drug Designation is an important regulatory advancement and the designation is granted to drugs that demonstrate promise for the treatment of a rare disease that affects fewer than 200,000 people in the U.S. Orphan designation is assigned based on the FDA’s evaluation of scientific and clinical data for the drug. Upon the FDA’s review of the data for SCI’s investigational product, the FDA has identified the drug as a promising treatment for sickle cell disease. The designation of Orphan status is granted to further advance the scientific development of promising medicinal products for rare diseases.
Orphan Drug Designation qualifies SCI for various development incentives.
Expanded Access Policy
SCI currently does not provide investigational product outside of clinical trials.
SCI is currently conducting a clinical trial on SC411, see clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT02973360?term=sancilio&rank=2
The investigational product, SC411, is currently not being provided for expanded access use.