Rare Disease Report

Hemophilic Artropathy Treatment Granted Orphan Drug Designation

NOVEMBER 21, 2017
Mathew Shanley
Tremeau Pharmaceuticals, Inc., a new pharmaceutical company focused on providing non-opioid treatment options for rare diseases, was granted orphan drug designation this morning for TRM-201 (rofecoxib), intended for the treatment of degenerative joint disease in patients with hemophilia, or hemophilic arthropathy (HA).

TRM-201 is a highly potent COX-2 selective non-steroidal anti-inflammatory drug (NSAID), and has a well-established efficacy profile. It is the first and only product to have been granted orphan drug designation for the treatment of 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 in a press release. “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.”

HA is a degenerative joint disease that occurs in patients with hemophilia. It is caused by recurrent intra-articular bleeding, and, according to a study in Haemophilia, is the largest cause of morbidity in this patient population. Both Hemophilia A (factor VIII deficiency) and B (factor IX deficiency) are genetic conditions and considered rare, as an estimated total of 20,000 cases have been diagnosed in the United States.

In controlled trials, rofecoxib has exhibited the ability to reduce the risk of gastrointestinal (GI) bleeding versus a traditional NSAID.

Rofecoxib is a non-narcotic analgesic and has no effect on bleeding time relative to placebo, however, like other NSAIDs, can cause an increased risk of serious cardiovascular thrombotic events, including severe myocardial infarction and stroke. It can also cause an increased risk of GI-related adverse events (AEs) including bleeding, ulceration, and perforation of the stomach or intestines.

“I avoid using traditional NSAIDs in my hemophilia patients due to their adverse effect on platelet function,” comments Stacy Croteau, MD, 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.”

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