Market Overview

Alnylam Announces that the United Kingdom's MHRA Grants Early Access to Patisiran


– Positive Scientific Opinion Under Early Access to Medicines Scheme
will Make Patisiran Treatment Available for UK hATTR Amyloidosis
Patients Before Marketing Authorization –

– Patisiran Indicated Within EAMS for the Treatment of Adults with
hATTR Amyloidosis –

Pharmaceuticals, Inc.
(NASDAQ:ALNY), the leading RNAi therapeutics
company, announced today that the UK's Medicines and Healthcare Products
Regulatory Agency (MHRA) has granted patisiran, an investigational RNAi
therapeutic in development for the treatment of hATTR amyloidosis, a
positive scientific opinion through the Early Access to Medicines Scheme
(EAMS). With this decision, eligible adults in the UK with hATTR
amyloidosis can gain access to patisiran before the drug is granted
marketing authorization by the European Commission (EC).

The aim of EAMS is to provide early availability of innovative, new,
unlicensed medicines to UK patients who have a high degree of unmet
clinical need. The medicines included in the scheme are those that are
intended to treat, diagnose or prevent seriously debilitating or
life-threatening conditions where there are no adequate treatment

"The EAMS positive scientific opinion reflects Alnylam's commitment to
patients with hATTR amyloidosis and their families, for whom it will be
welcome news," said Brendan Martin, Country Manager, UK & Ireland at
Alnylam. "New treatment options that impact the underlying cause of the
disease, improve neuropathy and a patient's ability to function on a
daily basis, are urgently needed and this decision will allow patients
to have access to patisiran without delay."

The MHRA's decision is based on the evaluation of the effects of
patisiran in hATTR amyloidosis patients with polyneuropathy and its
safety profile as demonstrated in the APOLLO Phase 3 study. The results
of the APOLLO study were published July 5, 2018 in The New
England Journal of Medicine
(NEJM). Within EAMS, patisiran will be
made available for eligible hATTR amyloidosis patients presenting with
symptoms of polyneuropathy and/or cardiomyopathy.

MHRA's decision follows the recent positive opinion by the CHMP for
patisiran for the treatment of hATTR amyloidosis in adult patients with
stage 1 or stage 2 polyneuropathy, with the EC decision expected in
September. If approved by the EC, the medicine will be commercialized
under the brand name ONPATTRO™. Patisiran is currently under priority
review as a Breakthrough Therapy with the U.S. Food and Drug
Administration (FDA), with an action date of August 11, 2018. Regulatory
filings in other markets, including Japan, are planned starting in

About Patisiran

Patisiran is an investigational, intravenously administered RNAi
therapeutic targeting transthyretin (TTR) in development for the
treatment of hereditary ATTR amyloidosis. It is designed to target and
silence specific messenger RNA, potentially blocking the production of
TTR protein before it is made. This may help to reduce the deposition
and facilitate the clearance of TTR amyloid in peripheral tissues and
potentially restore function to these tissues. Patisiran has not been
approved by the European Commission, the U.S. Food and Drug
Administration, or any other regulatory authority and does not yet have
marketing authorization.

About hATTR Amyloidosis

Hereditary TTR-mediated amyloidosis (hATTR) is an inherited,
progressively debilitating, and often fatal disease caused by mutations
in the TTR gene. TTR protein is primarily produced in the liver and is
normally a carrier of vitamin A. Mutations in the TTR gene cause
abnormal amyloid proteins to accumulate and damage body organs and
tissue, such as the peripheral nerves and heart, resulting in
intractable peripheral sensory neuropathy, autonomic neuropathy, and/or
cardiomyopathy, as well as other disease manifestations. hATTR
amyloidosis represents a major unmet medical need with significant
morbidity and mortality, affecting approximately 50,000 people
worldwide. The median survival is 4.7 years following diagnosis, with a
reduced survival (3.4 years) for patients presenting with
cardiomyopathy. Treatment options that can modify the course of the
disease are limited and there remains a pressing need for novel
medicines to help treat patients with hATTR amyloidosis.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing
that represents an extremely promising and rapidly advancing frontier in
biology and drug development today. Its discovery has been heralded as a
major scientific breakthrough that happens once every decade or so and
was recognized with the award of the 2006 Nobel Prize for Physiology or
Medicine. By harnessing the natural biological process of RNAi occurring
in our cells, a major new class of medicines, known as RNAi
therapeutics, is on the horizon. Small interfering RNA (siRNA), the
molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic
platform, function upstream of today's medicines by potently silencing
messenger RNA (mRNA) – the genetic precursors that encode for
disease-causing proteins - thus preventing them from being made. This is
a revolutionary approach with the potential to transform the care of
patients with genetic and other diseases.

About Alnylam

Alnylam (NASDAQ:ALNY) is leading the translation of RNA interference
(RNAi) into a whole new class of innovative medicines with the potential
to transform the lives of people afflicted with rare genetic,
cardio-metabolic, and hepatic infectious diseases. Based on Nobel
Prize-winning science, RNAi therapeutics represent a powerful,
clinically validated approach for the treatment of a wide range of
severe and debilitating diseases. Founded in 2002, Alnylam is delivering
on a bold vision to turn scientific possibility into reality, with a
robust discovery platform and deep pipeline of investigational
medicines, including four product candidates that are in late-stage
development. Looking forward, Alnylam will continue to execute on its
"Alnylam 2020" strategy of building a multi-product, commercial-stage
biopharmaceutical company with a sustainable pipeline of RNAi-based
medicines to address the needs of patients who have limited or
inadequate treatment options. Alnylam employs over 800 people in the
U.S. and Europe and is headquartered in Cambridge, MA.

Alnylam Forward Looking Statements

Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including, without limitation,
Alnylam's views with respect to the positive scientific opinion issued
by EAMS, data supporting the CHMP positive opinion, and ongoing
regulatory reviews of patisiran, planned regulatory filings, and
expectations regarding its "Alnylam 2020" guidance for the advancement
and commercialization of RNAi therapeutics, constitute forward-looking
statements for the purposes of the safe harbor provisions under The
Private Securities Litigation Reform Act of 1995. Actual results and
future plans may differ materially from those indicated by these
forward-looking statements as a result of various important risks,
uncertainties and other factors, including, without limitation,
Alnylam's ability to discover and develop novel drug candidates and
delivery approaches, successfully demonstrate the efficacy and safety of
its product candidates, the pre-clinical and clinical results for its
product candidates, which may not be replicated or continue to occur in
other subjects or in additional studies or otherwise support further
development of product candidates for a specified indication or at all,
actions or advice of regulatory agencies, which may affect the design,
initiation, timing, continuation and/or progress of clinical trials or
result in the need for additional pre-clinical and/or clinical testing,
delays, interruptions or failures in the manufacture and supply of its
product candidates, obtaining, maintaining and protecting intellectual
property, Alnylam's ability to enforce its intellectual property rights
against third parties and defend its patent portfolio against challenges
from third parties, obtaining and maintaining regulatory approval,
pricing and reimbursement for products, progress in establishing a
commercial and ex-United States infrastructure, competition from others
using technology similar to Alnylam's and others developing products for
similar uses, Alnylam's ability to manage its growth and operating
expenses, obtain additional funding to support its business activities,
and establish and maintain strategic business alliances and new business
initiatives, Alnylam's dependence on third parties for development,
manufacture and distribution of products, the outcome of litigation, the
risk of government investigations, and unexpected expenditures, as well
as those risks more fully discussed in the "Risk Factors" filed with
Alnylam's most recent Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission (SEC) and in other filings that
Alnylam makes with the SEC. In addition, any forward-looking statements
represent Alnylam's views only as of today and should not be relied upon
as representing its views as of any subsequent date. Alnylam explicitly
disclaims any obligation, except to the extent required by law, to
update any forward-looking statements.

Patisiran has not been approved by the European Commission, the U.S.
Food and Drug Administration, or any other regulatory authority.

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