Continues Collaboration to Provide Regulatory Submission Support for
Additional Patisiran Submissions
SOUTHBOROUGH, MA – August 20, 2018 – Veristat, a full service Clinical Research Organization (CRO), congratulates Alnylam on the recent FDA approval of ONPATTRO™ (patisiran), a lipid complex injection for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
“On behalf of the entire Veristat team that has supported Alnylam in the development of this therapy, we are humbled to have contributed towards this first-in-class drug approval for Alnylam, for the patients with hATTR amyloidosis and their families,” said John P. Balser, Ph.D., President and Co-Founder of Veristat. “We are continuing to support Alnylam as they look to expand regulatory approval of this treatment to other regions of the world.”
Veristat began working with Alnylam over 11 years ago and helped design the studies and prepare the Investigational New Drug (IND) submission for patisiran. Our collaboration continued throughout the entire patisiran program inclusive of a phase 2 study, a phase 3 pivotal study, and a phase 3 open-label extension study. Our teams have provided Alnylam with biostatistics & programming, data analysis and conversion, Data Safety Monitoring Board (DSMB) coordination, medical writing and project management support for these trials. Currently, Veristat is assisting Alnylam with the ongoing open-label extension and the preparation of their patisiran regulatory submissions for the Pharmaceutical and Medical Devices Agency (PMDA) in Japan and other health agencies.
“I am grateful for the dedication, excitement, and clinical trial expertise that the Veristat team has given Alnylam since the start of this program,” said Akshay Vaishnaw, MD, PhD, President of Research & Development at Alnylam. “Veristat accepted our challenge and their collaboration has been critical to the successful clinical trials that led to the U.S. FDA approval of ONPATTRO.”
Veristat is focused, dedicated and experienced in helping bring new therapies through the clinical development and regulatory submission process for patients who have no existing or limited treatment options available. Over 30% of the clinical trials and nearly 40 submission projects that our teams have supported are for rare disease indications like the treatments researched by Alnylam.
“The Veristat team has been an enthusiastic partner with Alnylam for over 11 years. We are thrilled to have supported the success of the clinical development programs at Alnylam,” stated Patrick Flanagan, Chief Executive Officer at Veristat. “I congratulate the entire Alnylam team on the landmark FDA approval of ONPATTRO.”
Important Safety Information About ONPATTRO:
Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.
To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended Supplementation
ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.
Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).
The most common adverse reactions that occurred in patients treated with ONPATTRO were upper respiratory tract infections (29%) and infusion related reactions (19%).
For additional information about ONPATTRO, please see the full Prescribing Information.
Veristat is a clinical research partner focused on supporting biopharmaceutical firms as they advance their therapies through the clinical development and regulatory submission process. Our work delivers meaningful clinical impact and our regulatory submission expertise is unrivaled in our industry. Veristat teams have worked on over 70 regulatory submission projects that have resulted in more than 40 submission approvals to date from various regulatory agencies around the world. Over the last 5 years alone, nearly 8% of new molecular entities (NMEs) approved by the FDA were supported by Veristat teams. Veristat aims to propel even the most complex submissions to successful regulatory conclusions so that new therapies become available to improve and save lives. For more information, visit www.veristat.com.
Gillian Dellacioppa, Marketing Director
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