dm+d

Unassigned

New Medicines

Loargys Arginase 1 deficiency

Information

Loargys
New molecular entity
Immedica Pharma
Immedica Pharma

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Recombinant enzyme replacement derived from human arginase 1 and engineered to include the substitution of cobalt for the manganese cofactor and pegylated to increase catalytic activity and serum stability compared to native human arginase 1
ARG1-D has been estimated to occur in 1 in 300,000-1,000,000 births. It is among the least common of all the disorders of the urea cycle. The estimated frequency of urea cycle disorders collectively is one in 30,000. However, because urea cycle disorders like ARG1-D often go unrecognized, these disorders are under-diagnosed, making it difficult to determine the true frequency of urea cycle disorders in the general population [1]. Company estimates there are 25 people with ARG1-D in the UK [4].
Arginase 1 deficiency
Subcutaneous injection