FDA grants orphan drug designation to ChemiCare’s drug candidate
CIC-39, ChemiCare‘s drug candidate, has received orphan drug designation from the Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy, a rare and fatal genetic disorder for which no definitive cure currently exists.
Affiliated and incubated by Bio4Dreams since 2021, the innovative SME develops modulators of Store-Operated Calcium Entry (SOCE) as therapies for calcium-related diseases.
After the EMA, another milestone for ChemiCare
This achievement marks a significant step in ChemiCare’s growth, following the company’s 2022 receipt of two orphan drug designations from the European Medicines Agency (EMA) one for Duchenne muscular dystrophy and another for other severe myopathies. Orphan drug designation not only offers financial incentives but also encourages the development of innovative therapies for conditions that currently lack adequate treatments.
ChemiCare is currently completing preclinical safety and tolerability studies, with the goal of initiating clinical trials by 2026. This step is crucial to ensure the candidate drug is safe for human use, and ChemiCare is making steady progress toward this objective.
Profile of the drug candidate CIC-39
CIC-39, along with other molecules developed by ChemiCare, has high bioavailability and a favorable toxicity profile that allows for chronic oral administration.
Additionally, thanks to its mechanism of action, it is expected that CIC-39 could also be used to treat other chronic diseases related to intracellular calcium imbalances, including several autoimmune disorders such as multiple sclerosis and systemic lupus erythematosus.
In this context, ChemiCare is positioning itself as a key player in the development of new therapies for rare diseases, and the success of CIC-39 could offer a valuable therapeutic solution for patients who currently lack adequate treatment options.