TRM-201 (rofecoxib) is an investigational treatment being developed by Tremeau Pharmaceuticals for severe joint disease in patients with hemophilia. Known as hemophilic arthropathy, or permanent joint disease, it is a long-term consequence of repeated hemarthrosis, or bleeding into joints, and can be common in hemophilia patients.

Hemophilic arthropathy is considered an orphan disease, meaning that it is classified as a rare condition affecting only small numbers of individuals (fewer than 200,000 people, according to the definition of a rare disease in the U.S.).

No medication is currently approved in the U.S. to treat hemophilic arthropathy.

How TRM-201 works

TRM-201 is a non-steroidal anti-inflammatory drug (NSAID), which are medicines used to reduce inflammation, relieve pain and lower body temperature. NSAIDs act by blocking two enzymes in the body, called cyclooxygenase (COX)-1 and -2, which cause inflammation, pain, and fever, and also help in blood clotting and in protecting the stomach from the damaging effects of stomach acid.

TRM-201 binds to and inhibits COX-2. As a result, it aims to reduce inflammation, pain and fever, and also prevents the clotting of blood (anti-platelet effect) and reduces gastrointestinal bleeding compared to other NSAIDs.

TRM-201 in development

TRM-201 in in November 2017 became the first and, to date, only medication designated an orphan drug by the U.S. Food and Drug Administration (FDA) for the treatment of hemophilic arthropathy. This is a special status given to a potential treatment for a rare disease, and is intended to speed its development.

A clinical development plan for TRM-201 has been approved with the FDA and the company is beginning work to test TRM-201 in clinical trials as a potential treatment of hemophilic arthropathy, and possibly for other orphan diseases in the future.

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