News from the FDA/CDC

FDA grants orphan drug status to rofecoxib for hemophilic arthropathy


The Food and Drug Administration on Nov. 21 granted orphan drug designation to rofecoxib (TRM-201), a cyclooxygenase 2–selective nonsteroidal anti-inflammatory drug (NSAID) intended to treat patients with hemophilic arthropathy (HA).

HA, a joint disease caused by hemarthrosis, is the largest cause of morbidity for hemophilia patients. There are currently no approved treatments in the United States.

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Rofecoxib was previously approved in the United States in 1999 under the brand name Vioxx, for treatment of arthritis and acute pain. In 2004, Merck voluntarily withdrew the drug over concerns about increased risk of myocardial infarction and stroke associated with long-term use.

The attempt at a reintroduction of rofecoxib specifically for the treatment of HA is being developed by Tremeau Pharmaceuticals.

Patients with hemophilia look to avoid traditional NSAIDs, as those drugs risk gastrointestinal ulcers and impair platelet aggregation. The current standard of care for HA is opioid treatment.

Rofecoxib and other NSAIDs cause an increased risk of serious cardiovascular thrombotic events and gastrointestinal adverse events.

Orphan drug status is available to treatments for rare disorders and provides a 7-year marketing exclusivity period against competition, along with tax credits and a waiver of Prescription Drug User Fee Act filing fees.

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