CMC Contrast AB, a privately owned drug development company that is developing a targeted contrast agent for Magnetic Resonance Imaging (MRI), announced today that Orphan Drug Designation has been granted by the US Food and Drug Administration (FDA) for its 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.
Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to novel drugs intended to treat a rare disease or condition affecting fewer than 200,000 people in the U.S. The designation confers special incentives to the drug developer, including tax credits towards the cost of clinical trials, prescription drug user fee waivers and may entitle a period of seven years U.S market exclusivity upon FDA approval.
Magnus Corfitzen, Chairman of CMC Contrast AB "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"
The FDA decision to grant the Orphan Drug Designation to CMC-001 is an important milestone for CMC Contrast AB. The company is now planning a pivotal phase III clinical program which in conjunction with the completed phase I and II clinical studies should enable CMC-001 to obtain regulatory approval and marketing authorization in the US. 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.