Orphan Medical regulatory update

The Minneapolis company submitted its first NDA to the FDA for Cystadane (betaine anhydrous powder), to treat homocystinuria, a rare genetic disorder caused by errors of methionine metabolism.

The drug, a naturally occurring substance found in the body, reduces elevated homocysteine blood levels, which can lead to developmental and mental retardation, early thromboembolism, skeletal abnormalities, and demyelinization, seizure and psychiatric disorders, as well as premature death. ...