Jacksonville, FL and Irvine, CA – March 11, 2019 – Espero BioPharma, Inc., a pharmaceutical company focused on the development of therapeutics for unmet needs in thrombosis and cardiac rhythm control, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for tecarfarin for the prevention of systemic thromboembolism of cardiac origin in patients with End Stage Renal Disease (ESRD) and Atrial Fibrillation (AFib). Tecarfarin is a Phase III-ready, vitamin K antagonist taken orally once a day as an anticoagulant.
“Orphan Drug Designation underscores the importance of tecarfarin as a potential new therapy for managing patients with ESRD and AFib, a population with substantial unmet medical need,” said Quang Pham, Chairman and Chief Executive of Espero BioPharma. “We are pleased that this designation will facilitate the clinical development of tecarfarin and potentially allow us to bring this investigative therapy to patients more quickly.”
The FDA grants orphan status to drugs that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. The ODD program provides a drug developer with certain benefits and incentives, including a seven-year period of U.S. marketing exclusivity from the date of marketing authorization, waiver of FDA user fees, and tax credits for clinical research. The granting of an Orphan Drug Designation does not alter the FDA’s regulatory requirements to establish safety and effectiveness of a drug through adequate and well-controlled studies to support approval and commercialization.
The presence of either chronic kidney disease (CKD) or AFib, increases the risk of serious thromboembolic adverse clinical outcomes, such as stroke and death. Antithrombotic therapy is typically recommended to decrease this risk in AFib patients, but there are no approved therapies for patients with ESRD and AFib. Patients with ESRD and AFib have typically been excluded from clinical trials because the approved therapies for AFib have metabolic profiles that may increase drug exposures thereby increasing the known risks and challenges in managing these patients.
Tecarfarin is a Phase III-ready, vitamin K antagonist taken orally once a day as an anticoagulant like warfarin. Tecarfarin was designed using a retrometabolic drug design process and targets a metabolic pathway that is distinct from the one targeted by the most commonly prescribed anticoagulants. This gives tecarfarin a better safety profile and potentially eliminates specific side effect while maintaining or improving efficacy.
About Espero BioPharma, Inc.
Espero is a pharmaceutical company focused on the development of therapeutics for unmet needs in thrombosis and cardiac rhythm control. The Company is developing tecarfarin for use in patients requiring anticoagulation, budiodarone for the treatment of refractory AFib and a third novel candidate to treat arrhythmias. Our product candidates were designed using a retrometabolic drug design process which enables them to target a different metabolic pathway than the pathway targeted by the most commonly prescribed anticoagulants and drugs for the treatment of AFib in order to potentially eliminate specific side effects while maintaining or improving effectiveness.
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