The European Medicines Administration (EMA) granted ODD status to CER-001 for the treatment of LCATdeficiency in July 2021. Positive clinical results from CER-001 in LCAT disease have previously been published. In the Annals of Internal Medicine in March 2021, a case study of a patient who was about to undergo dialysis due to the rapid decline in renal function was described. The patient was able to avoid the need for dialysis during her treatment with CER-001 and in addition, lipid deposits in her corneas which had caused significant visual blurring, improved with treatment. The improvement in visual function was still observed after 1 year of follow-up. A second case was described in the Journal of Internal Medicine in November 2021 and showed that CER-001 reduced glomerular lipid deposits and slowed the patient’s decline in renal function. Furthermore, CER-001 remodeled his plasma lipoproteins by reducing the level of LpX, large abnormal lipid complexes known to be renally toxic. Orphan-drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the U.S. Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. The designation is granted based on the mechanism of action of the drug or biologic taken into consideration with the pathogenesis of the disease or condition, its course and prognosis as well as the availability of treatments and/or resistance to available treatments.
Orphan drug designation qualifies sponsors for incentives including: tax credits for qualified clinical trials, exemption from user fees, and a potential for seven years of market exclusivity after approval.