EDSIVO for Vascular Ehlers-Danlos Syndrome. PDUFA date under priority review June 25, 2019.
Back on December 26, 2018, Acer Therapeutics announced that the U.S. Food and Drug Administration (FDA) accepted for review Acer’s New Drug Application (NDA) for EDSIVO™ for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. The FDA also granted a priority review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. Priority review is a designation granted by the FDA to accelerate the review process for drugs that offer a significant improvement in treatment or provide treatment where no satisfactory alternative therapy exists.
“The acceptance of our NDA for EDSIVO™ is an important step in our efforts to help patients with vEDS, who suffer with a devastating disease that currently has no approved treatment,” said William Andrews, M.D., FACP, Chief Medical Officer of Acer. “We have had the honor of learning about the significant challenges of living with vEDS directly from patients and their families. This has in large part driven the hard work, passion and complete dedication that our small team has given to this effort, and we will continue to do so as the FDA reviews our NDA for EDSIVO™. We are excited about the possibility of making EDSIVO™ available in the U.S. for patients in the near future.”
“We continue to accelerate our pre-commercial activities supporting the potential U.S. launch of EDSIVO™ for the treatment of vEDS if it is approved by the FDA,” said Chris Schelling, CEO and Founder of Acer. “Additionally, we are working diligently on advancing and expanding our pipeline with the goal of bringing multiple products to patients with serious rare diseases over the next several years.”
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 U.S. 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 U.S. 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(1). FDA granted a priority review of the EDSIVO™ NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. EDSIVO™ received FDA Orphan Drug Designation for the potential treatment of vEDS in 2015. For more information, visit www.acertx.com.
Avatrombopag for Immune Thrombocytopenic Purpura (ITP). PDUFA date for sNDA filing June 30, 2019.
Back on November 5, 2018, Dova Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) accepted for review the supplemental New Drug Application (sNDA) for DOPTELET (avatrombopag) for a new indication, the treatment of chronic immune thrombocytopenia (ITP) in patients who have had an insufficient response to a previous treatment. ITP is an autoimmune bleeding disorder characterized by thrombocytopenia, i.e., an abnormally low level of platelets. The Prescription Drug User Fee Act (PDUFA) goal date for an FDA decision on the sNDA is June 30, 2019.
DOPTELET, a second generation, orally administered thrombopoietin receptor agonist (TPO-RA), was approved by FDA in May 2018 for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure.
“Chronic ITP affects approximately 60,000 adults in the United States and despite the currently available therapies, which include two other TPO-RAs, there remains an important unmet need,” said Lee F. Allen, MD, PhD and Chief Medical Officer of Dova. “Acceptance of this sNDA is another significant milestone for Dova, and an important step towards addressing this underserved patient population and expanding the applications for DOPTELET as a treatment for thrombocytopenia. We look forward to working closely with the FDA as they review this sNDA.”
The ITP sNDA is supported by safety and efficacy data from one pivotal randomized, placebo-controlled Phase 3 clinical trial in the target indication that met its primary (number of weeks with a platelet count ≥50×109/L in the absence of rescue therapy) and first secondary (proportion of subjects with platelet counts ≥50×109/L on Day 8) efficacy endpoints with high statistical significance (P<0.0001). Data from the Phase 3 clinical trial has been recently published online (Br J Haematol. 2018 Sep 7. doi: 10.1111/bjh.15573. [Epub ahead of print]), and will be included in an upcoming volume of the British Journal of Haematology. Additional supportive efficacy data for the ITP sNDA are provided by two Phase 2 ITP clinical trials, as well as the two Phase 3 trials for the treatment of thrombocytopenia in patients with CLD. Data from all 24 studies in the avatrombopag clinical development program support the safety and tolerability of avatrombopag across multiple indications.