October 2024 – Genewity is proud to announce that earlier this year, i-Thymus has been granted Orphan Drug Designation by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) for the treatment of congenital athymia. This recognition from both regulatory bodies marks an important milestone for the development of i-Thymus and highlights its potential to address a critical unmet medical need.
Acceleration forGenewity
The Orphan Drug Designation brings several key benefits that will accelerate the development of i-Thymus. Most significantly, it grants market exclusivity for up to ten years in Europe and seven years in the U.S. following approval, ensuring a strong competitive position in both markets. Genewity will also gain financial incentives, including reduced regulatory fees in the EU and tax credits for clinical trials in the U.S., which will help lower development costs and extend the company's resources.
In addition, both the EMA and FDA offer regulatory support to companies developing orphan drugs. This includes personalised guidance on clinical trial design and the overall regulatory pathway, reducing risks and increasing the likelihood of approval. This support will be critical as Genewity advances i-Thymus toward manufacturing under Good Manufacturing Practices (GMP) and investigations at clinical stage.
Acceleration for patients with congenital athymia
Congenital athymia, as seen in patients with complete DiGeorge syndrome, currently has limited treatments available. One, for example, is used in the U.S. but depends on the availability of donor tissue, which limits its accessibility and effectiveness for many patients. Genewity’s i-Thymus is designed to tackle the underlying cause of the disease at a genetic level with an autologous and potentially curative approach. By modifying a patient’s own cells to create functional autologous thymus tissue, i-Thymus could bring a transformative therapy to patients who urgently need it, providing them with a chance to develop a functioning immune system and significantly improving their quality of life.
What is next for Genewity
“Securing orphan designation in both Europe and the U.S. is a huge achievement for our small, ambitious team,” said Dr. Arno Bisschop, CEO of Genewity. “This validation strengthens our confidence in the potential of i-Thymus to make a real difference in the lives of patients without a functioning thymus. We are eager to move forward and take the next steps toward clinical development.”
With the Orphan Drug Designations in place, Genewity will be able to advance i-Thymus more quickly towards the clinic. Having the support of the EMA and FDA, i-Thymus is optimally positioned to create significant value for patients.
About Genewity
Genewity B.V.is a biotechnology company based at the Leiden Bioscience Park, Netherlands, specialising in the development of advanced treatments using extracorporeal gene therapy with lentiviral vectors. This technology enables the modificationof stem cell characteristics, opening new avenues for innovative gene-modified cell therapies. The company focuses on generating thymus cells from pluripotent stem cells to develop treatments for congenital athymia and autoimmune diseases, as well as restoring bone marrow stem cell function in patients with RAG-SCID.
