Tremeau Pharmaceuticals announced the U.S. Food and Drug Administration (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.”
Hemophilic arthropathy (HA) is a degenerative joint disease occurring in patients with hemophilia and is caused by recurrent intra-articular bleeding. It is the largest cause of morbidity in patients with hemophilia. No medications are currently approved or licensed in the United States to treat HA. Due to their chronic hypo-coagulability, patients with hemophilia are at a heightened risk for hemorrhaging events, including gastrointestinal bleeding. Traditional NSAIDs are avoided in this population due to their effects on platelet aggregation and risk of gastrointestinal ulcers, and high potency opioids are the current standard of care in treating HA.
TRM-201 (rofecoxib) is a highly potent COX-2 selective non-steroidal anti-inflammatory drug (NSAID) with a well-established efficacy profile and is the first and only product granted orphan designation status for the treatment for hemophilic arthropathy. Rofecoxib is a non-narcotic analgesic, has no effect on bleeding time relative to placebo and is the only COX-2 selective NSAID ever approved in the U.S. to demonstrate a reduced risk of gastrointestinal bleeding versus a traditional NSAID in a controlled trial.
Nonsteroidal anti-inflammatory drugs (NSAIDs), including rofecoxib, cause an increased risk of serious cardiovascular thrombotic events, including myocardial infarction and stroke, which can be fatal. This risk may occur early in treatment and may increase with duration of use. NSAIDs, including rofecoxib, are contraindicated in the setting of coronary artery bypass graft (CABG). NSAIDs, including rofecoxib, cause an increased risk of serious gastrointestinal (GI) adverse events including bleeding, ulceration, and perforation of the stomach or intestines, which can be fatal. These events can occur at any time during use and without warning symptoms. Elderly patients and patients with a prior history of peptic ulcer disease and/or GI bleeding are at greater risk for serious GI events.