CAMBRIDGE, Mass.--(BUSINESS WIRE)--Tremeau Pharmaceuticals, Inc. (Tremeau), a new pharmaceutical company focused on providing non-opioid pain treatments for rare diseases, announced today that it has recently gained agreement with the U.S. Food and Drug Administration (FDA), in a formal Type B Meeting, on the development requirements to support a New Drug Application (NDA) for TRM-201 (rofecoxib). Rofecoxib is a cyclooxygenase-2 (COX-2) selective non-steroidal anti-inflammatory drug (NSAID) recently granted an orphan drug designation (ODD) as a non-opioid pain alternative for the treatment of hemophilic arthropathy (HA).
“Aligning with the FDA on the phase III program size, duration, and dosing as well as the pharmacokinetic requirements is a major step forward for patients with HA and Tremeau,” explains Mark H.N. Corrigan, M.D., President of R&D for Tremeau, “The significant unmet medical need, and the known efficacy profile of rofecoxib enabled alignment on a streamlined program, including a single phase III study, that, if successful, will inform the safe and effective use in the HA patient population.”
“Combined with our recently approved orphan drug designation for the treatment of HA, gaining agreement with and guidance from FDA on an efficient development program for rofecoxib is an important milestone,” added Bradford C. Sippy, Chief Executive Officer of Tremeau, “We look forward to initiating our phase III study for rofecoxib and bringing forth a viable alternative to opioids for patients with HA.”
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
TRM-201 (rofecoxib) is a highly potent cyclooxygenase-2 (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.
Tremeau plans to develop and commercialize rofecoxib for the orphan indication of the treatment of hemophilic arthropathy and subsequent niche patient segments with no approved treatments and where it could have a strong benefit to risk ratio versus other therapies.
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 [6].
About Tremeau Pharmaceuticals, Inc.
Tremeau is a new pharmaceutical company focused on providing non-opioid pain treatments 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.
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).
6. US Food and Drug Administration. Developing Products for Rare Diseases & Conditions. https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/
