FDA grants orphan designation for Hemophilic Arthropathy treatment TRM-201
Tremeau Pharmaceuticals, a new pharmaceutical company focused on providing non-opioid pain treatments for rare diseases, announced that the FDA has granted an orphan drug designation, on a first cycle review, for TRM-201 (rofecoxib), a COX-2 selective non-steroidal anti-inflammatory drug (NSAID), for the treatment of degenerative joint disease in patients with hemophilia, also known as hemophilic arthropathy (HA).
“Being granted an orphan drug designation for rofecoxib by FDA is an important regulatory milestone for Tremeau and affirms our strategy of providing non-opioid pain treatments for rare diseases like hemophilic arthropathy,” said Bradford C. Sippy, Chief Executive Officer of Tremeau. “Combined with our ongoing conversations with FDA of our proposed development plan for rofecoxib, we are well positioned to move forward with the development and introduction of rofecoxib for this specific use.”
“I avoid using traditional NSAIDs in my hemophilia patients due to their adverse effect on platelet function,” comments Stacy Croteau, M.D., MMS, Associate Director of the Boston Hemophilia Center and Attending Hematologist, Boston’s Children’s Hospital “With its well characterized, non-narcotic efficacy profile and lack of anti-platelet effect, rofecoxib is potentially well suited for patients suffering from hemophilic arthropathy.”
“Tremeau’s strategy of matching rofecoxib’s profile to specific populations who can benefit from the drug is a good approach,” added Professor Garret FitzGerald M.D. Chair, Department of Systems Pharmacology and Translational Therapeutics, University of Pennsylvania Perelman School of Medicine.