Marvel to File for Orphan Drug Designation with The U.S. FDA for MB-204 as a Treatment for Rett Syndrome
March 17, 2025 7:00 AM EDT | Source: Marvel Biosciences Corp.
Calgary, Alberta--(Newsfile Corp. - March 17, 2025) - Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc. (collectively the "Company" or "Marvel"), announced plans to file for Orphan Drug Designation (ODD) with the U.S. Food and Drug Administration (FDA) for its lead compound, MB-204, as a potential treatment for Rett Syndrome.
This decision follows strong preclinical data showing MB-204's sustained benefits, outperforming Trofinetide-the only FDA-approved treatment for the disease. Marvel aims to leverage ODD benefits to accelerate MB-204's clinical development and provide new hope to patients and families.
Marvel's preclinical study, conducted in collaboration with the iBraiN Institute, evaluated MB-204's efficacy using Trofinetide as a benchmark. Led by Drs. Julie le Merrer and Jerome Becker, the study assessed key behavioral outcomes in a Rett Syndrome mouse model (Mecp2 +/-), including social interaction, memory, and repetitive behaviors.
"We are very pleased with these results and excited to take the next step in seeking Orphan Drug Designation," said Dr. Mark Williams, Chief Science Officer of Marvel Biosciences. "Trofinetide, which received FDA approval in 2023 for Rett Syndrome, also holds Orphan Drug Status. Given the encouraging data from our study, we see a strong case for MB-204 to follow a similar regulatory path."
Orphan Drug Designation is a critical regulatory pathway that supports the development of treatments for rare diseases affecting fewer than 200,000 individuals in the U.S. Rett Syndrome, a severe neurodevelopmental disorder primarily affecting girls, has limited treatment options, making ODD an important tool for advancing new therapies. The designation provides key benefits, including market exclusivity, financial support through tax credits, waived FDA user fees, and access to expedited programs such as fast-track and priority review to accelerate the approval process.
"Orphan Drug Designation will help validate MB-204's potential in Rett Syndrome and reinforce Marvel's position as a leader in rare neurological therapies," said Rod Matheson, CEO of Marvel Biosciences. "This designation would provide essential support as we work to bring this much-needed treatment to patients."
About Marvel Biosciences Corp.
Marvel Biosciences Corp., and its wholly owned subsidiary, Marvel Biotechnology Inc., is a Calgary-based pre-clinical stage pharmaceutical development biotechnology company. The Company is developing MB-204, a novel fluorinated derivative of the approved anti-Parkinson's drug Istradefylline, the only clinically approved adenosine A2a antagonist. A significant and growing body of scientific evidence suggests drugs that block the adenosine A2a receptor, such as MB-204, could be useful in treating other neurological diseases such as autism, depression and Alzheimer's Disease. The Company is actively investigating its potential in addressing other neurodevelopmental disorders, such as Rett Syndrome and Fragile X Syndrome, to expand its therapeutic reach.
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