Market Overview

Veristat Congratulates Alnylam on the FDA Approval of ONPATTRO™ (patisiran)


Continues Collaboration to Provide Regulatory Submission Support for
Additional Patisiran Submissions

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
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
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).

Adverse Reactions
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

About Veristat:

is a clinical research partner focused on supporting biopharmaceutical
firms as they advance their therapies through the clinical
and regulatory
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

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