The US Food and Drug Administration, FDA, has granted orphan drug designation for the company’s liver specific contrast agent CMC-001.
The Orphan Drug Designation has been granted for CMC-001 for use as a targeted contrast agent for diagnostic MRI for detection and localization of focal liver lesions in patients where gadolinium-based contrast agents are contraindicated or cannot be administered, according to a recent press release.
“The diagnostic benefits of enhanced MRI liver imaging in detecting and localizing focal liver lesions are well established. However, a small subset of patient groups cannot undergo enhanced liver MRI procedures due to the risks of severe side effects associated with currently used gadolinium based contrast agents. For these patients, including patients with severe chronic renal insufficiency, important diagnostic information may be missed due to lack of contrast enhanced MRI. CMC-001 will allow radiologists to obtain the best possible diagnostic information in these subsets of patients, enabling clinicians to select the optimal treatment option for these patients. This is not possible today as clinicians only have access to data from an unenhanced imaging procedure on which they will base their disease management decisions for these patients,” commented Magnus Corfitzen, Chairman of CMC Contrast AB.
CMC-001 is orally administered and consists of manganese combined with uptake promoters (manganese (II) chloride tetrahydrate (with L-alanine and vitamin D3 as promoters of absorption)).
The company states that the news form an important milestone for CMC Contrast and that it is now planning a pivotal phase III clinical program. In conjunction with the completed phase I and II clinical studies, this should enable CMC-001 to obtain regulatory approval and marketing authorization in the US. CMC Contrast states that CMC-001 will then become available to patients in need of a liver MRI procedure for whom gadolinium based contrast agents are contraindicated or cannot be administered.