Europe’s Committee for Orphan Medicinal Products issued a positive recommendation for orphan drug designation for LJPC-401, a formulation of synthetic human hepcidin, for the treatment of sickle cell disease (SCD).
The recommendation was based on the results of a phase I study of patients with hereditary hemochromatosis, thalassemia, or SCD who are at risk for developing iron overload. Fifteen patients received LJPC-401 at doses ranging from 1 to 20 mg. Treatment resulted in a statistically significant reduction in serum iron levels (p=0.008).
For those receiving 20 mg of LJPC-401, serum iron was reduced by an average of 58.1 percent from baseline to eight hours (p=0.001), and a reduction of 21.2 percent from baseline was maintained after seven days. Serum iron did not return to baseline levels after seven days.
The drug appeared to be well tolerated with no dose-limiting toxicities. The most common treatment-related adverse events were injection site reactions, which were mild to moderate, self-limiting, and fully resolved, according to the study.
A randomized, controlled, multicenter, pivotal study of LJPC-401 in patients with beta-thalassemia who are suffering from iron overload is planned for 2017.
LJPC-401 previously received orphan designation in Europe for patients with beta-thalassemia intermedia and major.
Source: La Jolla Pharmaceutical Company press release, October 27, 2016.