Acer Therapeutics Submits NDA for EDSIVO; Requests Priority Review
Acer Therapeutics Inc. recently announced it has submitted a New Drug Application (NDA) to the US FDA for EDSIVO for the treatment of vascular Ehlers-Danlos syndrome (vEDS). Pursuant to the Prescription Drug User Fee Act (PDUFA), the FDA has 60 days to determine whether to accept the submission for review. Along with the NDA submission, Acer has requested Priority Review, which if granted, could result in a six-month review period. Priority Review is a designation given to drugs that offer a significant improvement in treatment or provide treatment where no satisfactory alternative therapy exists.
“Our NDA submission represents the culmination of extensive efforts of our employees, investigators, clinical trial sites, contract research organizations, caregivers, and patients,” said William Andrews, MD, FACP, Chief Medical Officer of Acer. “We now look forward to continuing to work with the FDA as they review our NDA, with hopes to make EDSIVO available as quickly as possible in the US. We are grateful to the vEDS patient and advocacy community for their continued involvement, support and feedback as we work together to advance EDSIVO™, which has the potential to be a significant step forward in the care of patients with this devastating disease.”
Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders of connective tissue. vEDS is the most severe subtype where patients suffer from life threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures. The average mortality is 51 years of age. An Acer-commissioned patient-finder study phenotypically identified 4,169 vEDS patients in the US from an analysis of a commercially available patient claims database with data of approximately 190 million unique patient lives.
Based on that information, Acer estimates the prevalence of phenotypically defined vEDS in the US could be greater than 1 in 45,000. Currently, there are no FDA-approved therapies for vEDS. Acer is advancing EDSIVO (celiprolol), a new chemical entity (NCE), for the treatment of vEDS based on a randomized controlled clinical study of celiprolol and submitted an NDA to the FDA in October 2018. Acer requested priority review for EDSIVO which, if granted at the time of potential acceptance of NDA for filing, could result in a Prescription Drug User Free Act (PDUFA) action date of late second quarter 2019. EDSIVO received FDA Orphan Drug Designation for the potential treatment of vEDS in 2015.
Acer, headquartered in Newton, MA, is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for patients with serious rare and ultra-rare diseases with critical unmet medical need. Acer’s late-stage clinical pipeline includes two candidates for severe genetic disorders: EDSIVO (celiprolol) for vascular Ehlers-Danlos syndrome (vEDS), and ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate) for urea cycle disorders (UCD) and Maple Syrup Urine Disease (MSUD). There are no FDA-approved drugs for vEDS and MSUD and limited options for UCD, which collectively impact approximately 7,000 patients in the US. Acer’s product candidates have clinical proof-of-concept and mechanistic differentiation, and Acer intends to seek approval for them in the U.S. by using the regulatory pathway established under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act (FFDCA) that allows an applicant to rely at least in part on third-party data for approval, which may expedite the preparation, submission, and approval of a marketing application. For more information, visit www.acertx.com.
Total Page Views: 278