Photo by Steven Harbour
Prescription drugs
The US Food and Drug Administration (FDA) has granted orphan drug designation to rofecoxib (TRM-201) as a potential treatment for degenerative joint disease in hemophilia, also known as hemophilic arthropathy (HA).
Rofecoxib is a COX-2 selective non-steroidal anti-inflammatory drug (NSAID) that was previously sold in the US under the name Vioxx.
Vioxx was FDA-approved to relieve the signs and symptoms of osteoarthritis, manage acute pain in adults, and treat primary dysmenorrhea.
Merck & Co. pulled Vioxx from the US market in 2004 due to safety concerns. The drug was shown to increase a person’s risk of cardiovascular events, including heart attack and stroke.
Now, Tremeau Pharmaceuticals, Inc., is working to bring rofecoxib back to market to treat patients with HA.
HA patients should not receive traditional NSAIDs due to their effects on platelet aggregation and the risk of gastrointestinal ulcers associated with these drugs. Therefore, high potency opioids are the current standard of care in 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.
Sippy is a former Merck employee who helped with the recall of Vioxx and knew the final patent protecting the drug’s monopoly was expiring this fall.
When it stopped making Vioxx, Merck was facing thousands of lawsuits from people claiming the drug caused their heart attacks or strokes.
Merck’s own research showed the drug doubled those risks, but lawyers for patients claimed the company downplayed or concealed that. Merck initially fought the lawsuits but, in 2007, agreed to a $4.85 billion settlement.
If approved to treat HA, rofecoxib would carry a warning about the increased risk of heart attack and stroke associated with the drug.
Although the orphan designation for rofecoxib is a step toward FDA approval, Sippy said Tremeau must still raise $25 million or more to fund trials of the drug in hemophilia patients.
About orphan designation
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.
