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FDA Grants Orphan Drug Designation for TRM-201 (rofecoxib) for the Treatment of Hemophilic Arthropathy

FDA Grants Orphan Drug Designation for TRM-201 (rofecoxib) for the Treatment of Hemophilic Arthropathy

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CAMBRIDGE, Mass.–(BUSINESS WIRE) November 21, 2017 –Tremeau Pharmaceuticals, Inc., a new pharmaceutical company focused on providing non-opioid pain treatments for rare diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation, on a first cycle review, for TRM-201 (rofecoxib), a COX-2 selective non-steroidal anti-inflammatory drug (NSAID), for the treatment of degenerative joint disease in patients with hemophilia, also known as hemophilic arthropathy (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. “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.”

“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.”

“Tremeau’s strategy of matching rofecoxib’s profile to specific populations who can benefit from the drug is a good approach,” added Professor Garret FitzGerald M.D. Chair, Department of Systems Pharmacology and Translational Therapeutics, University of Pennsylvania Perelman School of Medicine.

About Hemophilic Arthropathy

Hemophilic arthropathy (HA) is a degenerative joint disease occurring in patients with hemophilia and is caused by recurrent intra-articular bleeding. It is the largest cause of morbidity in patients with hemophilia [1]. No medications are currently approved or licensed in the United States to treat HA. Due to their chronic hypo-coagulability, patients with hemophilia are at a heightened risk for hemorrhaging events, including gastrointestinal bleeding. Traditional NSAIDs are avoided in this population due to their effects on platelet aggregation and risk of gastrointestinal ulcers [2], and high potency opioids are the current standard of care in treating HA [3].

About TRM-201 (rofecoxib)

TRM-201 (rofecoxib) is a highly potent COX-2 selective non-steroidal anti-inflammatory drug (NSAID) with a well-established efficacy profile and is the first and only product granted orphan designation status for the treatment for hemophilic arthropathy. Rofecoxib is a non-narcotic analgesic, has no effect on bleeding time relative to placebo [4] and is the only COX-2 selective NSAID ever approved in the U.S. to demonstrate a reduced risk of gastrointestinal bleeding versus a traditional NSAID in a controlled trial [5].

Nonsteroidal anti-inflammatory drugs (NSAIDs), including rofecoxib, cause an increased risk of serious cardiovascular thrombotic events, including myocardial infarction and stroke, which can be fatal. This risk may occur early in treatment and may increase with duration of use. NSAIDs, including rofecoxib, are contraindicated in the setting of coronary artery bypass graft (CABG). NSAIDs, including rofecoxib, cause an increased risk of serious gastrointestinal (GI) adverse events including bleeding, ulceration, and perforation of the stomach or intestines, which can be fatal. These events can occur at any time during use and without warning symptoms. Elderly patients and patients with a prior history of peptic ulcer disease and/or GI bleeding are at greater risk for serious GI events.

About FDA Orphan Drug Designation Program

The FDA Orphan Drug Designation program provides orphan status to drugs intended to treat rare diseases or disorders that affect fewer than 200,000 people in the US. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain incentives, including federal grants, tax credits and a waiver of Prescription Drug User Fee Act filing fees.

About Tremeau Pharmaceuticals, Inc.

Tremeau is a new pharmaceutical company focused on providing non-opioid treatment options for rare diseases and other unique patient conditions with no approved treatments. Tremeau’s unique approach to acute and chronic pain in rare diseases is rooted in the mechanism of action, documented efficacy, and clinically differentiated profile of COX-2 selective NSAIDs.

Tremeau plans to develop and commercialize rofecoxib for the orphan indication of the treatment of hemophilic arthropathy and subsequent niche patient segments where it has a strong benefit to risk ratio versus other therapies.

For additional information about Tremeau, please visit www.tremeaurx.com.

References

1. Forsyth A, Gregory M, Nugent D, et al. Haemophilia Experiences, Results and Opportunities (HERO) Study: survey methodology and population demographics. Haemophilia 2014;20;44-51

2. Srivastava et al., Guidelines for the management of hemophilia. Haemophilia 2013; 19: e1–47

3. Witkop M, Lambing A, Divine G, Kachalsky E, Rushlow D, Dinnen J. A national study of pain in the bleeding disorders community: a description of haemophilia pain. Haemophilia 2012; 18: e115–19.

4. US Food and Drug Administration. VIOXX (Rofecoxib) U.S. Prescribing Information May 09, 2016, (access September 27, 2017)

5. US Food and Drug Administration. Analysis and recommendations for agency action regarding non-steroidal anti-inflammatory drugs and cardiovascular risk. http://www.fda.gov/Drugs/DrugSafety/PostmarketDrugSafetyInformationforPatientsandProviders/ (accessed September 27, 2017).

Source: Tremeau Pharmaceuticals, Inc.

Posted: November 2017

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