The European Commission (EC) has granted Orphan Medicinal Product Designation (OMPD) to Sangamo Therapeutics’ TX200, a wholly-owned autologous Chimeric Antigen Receptor Regulatory T Cell (CAR-Treg) cell therapy product candidate for treatment in solid organ transplantation.
“Patients who have received a solid organ transplant require lifelong surveillance and chronic immunosuppressive medications to manage the risk of transplant rejection,” said Rob Schott, MD, MPH, FACC, head of development for Sangamo. “Our goal with TX200 is to create a transformative therapy that reduces the risk of organ rejection, while reducing the patient burden from chronic immunosuppressive therapy. Achieving this important regulatory milestone takes us one step closer to realizing that goal for patients.”
Dosing of the first patient in the STEADFAST Phase 1/2 clinical study took place in March 2022, with the second patient dosing planned later this quarter.
The EC granted OMPD to TX200 following a positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products. To qualify for orphan designation, a treatment must be intended for a life-threatening or chronically debilitating disease affecting fewer than 5 in 10,000 people. Importantly, no satisfactory method of treatment must exist, or if such a method exists, the treatment must be of significant benefit to patients.
The OMPD status offers a range of incentives to encourage the development of orphan medicines, including protocol assistance on study protocols, potential fee reductions, and 10 years of market exclusivity upon regulatory approval.
