New Treatment for Hemophilic Joint Disease Granted Orphan Drug Status by FDA

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Fixx Pharmaceuticals

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to rofecoxib (TRM-201), an non-opioid investigational pain treatment for hemophilic arthropathy (HA, a hemophilia degenerative joint disease) under development by Tremeau Pharmaceuticals.

Rofecoxib is a COX-2 selective non-steroidal anti-inflammatory drug (NSAID) with a well-established efficacy profile. It is the first (and only) product created for the treatment of degenerative joint disease in patients with hemophilia.

The drug candidate is a non-narcotic analgesic with no effect on bleeding time relative to placebo and with a demonstrated reduced risk of gastrointestinal bleeding compared to traditional NSAIDs, according to the pharmaceutical company.

“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,” Bradford C. Sippy, chief executive officer of Tremeau, said 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,” he added.

HA occurs in patients with hemophilia and is a consequence of recurrent bleeding within the joints. There are currently no medications for HA that have been approved or licensed in the United States.

Because hemophilia patients have a decreased or deficient coagulability of their blood (hypo-coagulability), they are also at heightened risk for different types of hemorrhage, such as gastrointestinal (GI) bleeding.

Rofecoxib may also cause an increased risk of serious GI events, and it is not indicated for elderly patients and those with a past history of peptic ulcer disease and/or GI bleeding. Rofecoxib is not indicated in the setting of coronary artery bypass graft (CABG) due to the increased risk of cardiovascular thrombotic events, including myocardial infarction and stroke.

Due to these adverse effects and its impact on platelet aggregation, traditional NSAIDs have been avoided for HA patients, and high potency opioids have typically been used as the standard of care in the treatment of HA. But rofecoxib might change this scenario.

“I avoid using traditional NSAIDs in my hemophilia patients due to their adverse effect on platelet function,” said Dr. Stacy Croteau, MD, MMS, associate director of the Boston Hemophilia Center. “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 FDA grants orphan drug status to investigational treatments for rare diseases (conditions that affect fewer than 200,000 people in the U.S.) and provides up to seven years of market exclusivity, as well as incentives, tax credits, and waivers.