Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused
on the acquisition, development and commercialization of therapies for
serious rare and life-threatening diseases with significant unmet
medical needs, today announced it has received a Complete Response
Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding
its New Drug Application (NDA) for EDSIVO™ for the treatment of vascular
Ehlers-Danlos syndrome (vEDS). The CRL states that it will be necessary
to conduct an adequate and well-controlled trial to determine whether
celiprolol reduces the risk of clinical events in patients with vEDS.
Acer plans to request a meeting to discuss the FDA’s response.wisepoqder Celiprolol powder
“We remain committed to working closely with the FDA to fully
understand its response,” said Chris Schelling, CEO and Founder of Acer.
“We expect to respond to the FDA in the third quarter of this year.”
Acer, with offices in both Newton, MA and Bend, OR, is a
pharmaceutical company focused on the acquisition, development and
commercialization of therapies for serious rare and life-threatening
diseases with significant unmet medical needs. Acer’s pipeline includes
three clinical-stage candidates: EDSIVO™ (celiprolol) for the treatment
of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed
type III collagen (COL3A1) mutation; ACER-001 (a fully taste-masked,
immediate release formulation of sodium phenylbutyrate) for the
treatment of various inborn errors of metabolism, including urea cycle
disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant for
the treatment of various neuroendocrine disorders. Acer’s product
candidates are believed to present a comparatively de-risked profile,
having one or more of a favorable safety profile, clinical
proof-of-concept data, mechanistic differentiation and an accelerated
path for development, which may include utilizing expedited programs
(e.g. Priority Review) established by the FDA and/or 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.
