COUR Pharma has announced that their investigational therapy CNP-106 was granted Orphan Drug Designation for patients with generalized myasthenia gravis (gMG). Details were provided in a recent press release.
As the first therapy of its kind, CNP-106 is being developed to treat MG by directly targeting the immune response that triggers symptoms. It is a biodegradable drug that decreases levels of acetylcholine receptor (AChR) antibodies in patients with anti-AcHr positive gMG.
“Receiving orphan drug designation for CNP-106 is an important development and regulatory milestone for our myasthenia gravis program,” said President and Chief Executive Officer Dannielle Appelhans.
Orphan Drug Designation is granted by the U.S. Food and Drug Administration (FDA) to new therapies that hold promise for rare diseases that impact fewer than 200,000 individuals. The designation offers financial benefits and accelerates the regulatory process. If approved, for example, COUR Pharma will have several years of market exclusivity for CNP-106, preventing competitors from selling similar products.
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COUR Pharma is currently recruiting patients with anti-AChR positive gMG aged 18 to 75 for a new clinical trial to determine the safety and efficacy of CNP-106. Patients will be randomized to receive either a placebo or differing doses of CNP-106. They will then be followed to determine how patients tolerate the drug and what molecular effects it has on the body.
“As enrollment in our clinical trial for CNP-106 in gMG continues to advance, we are looking forward to sharing data readouts,” Appelhans stated.
In addition to their MG program, COUR Pharma is working to develop therapies for a number of autoimmune diseases by addressing the precise antibodies that cause the conditions. Among these are diabetes, Celiac disease and primary biliary cholangitis.
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