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FDA Grants Orphan Drug Designation for Rofecoxib, Pain Medication for Hemophilia Arthropathy

November 21, 2017
Orphan Drug Designation for Hemophila Pain Medication

The US Food and Drug Administration (FDA) has granted orphan drug designation to TRM-201 (rofecoxib), a class of COX-2 selective non-steroidal anti-inflammatory drug (NSAID) for the treatment of hemophilic arthropathy. Rofecoxib is manufactured by Cambridge, Massachusetts-based Tremeau Pharmaceuticals. Tremeau is focused on developing nonopioid pain treatments for rare diseases.

Rofecoxib is the chemical name for the drug formerly known as Vioxx, which was previously manufactured and marketed by Merck & Co. until it was voluntarily pulled by the company in 2004 in light of evidence that it doubled the chances of having a heart attack or stroke. The patent for Vioxx expired in the fall of 2017. 

Hematologists are often hesitant to prescribe NSAIDS for hemophilia patients as their use may increase bleeding risk, particularly for very serious internal bleeding.

“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.” 

The new FDA designation adds a potential benefit – the drug has not yet received FDA approval – for providers who often prescribe opioid painkillers to treat hemophilia patients grappling with chronic pain. Opiods, though effective, can come with their own set of drawbacks including risk for addiction.   

“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.”

Source: Tremeau press release and AP NewsBreak, dated November 21, 2017