Jul 27, 2018 Press Release for Alnylam
Alnylam Receives Positive CHMP Opinion for ONPATTRO™ (patisiran) for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis in Adults with Stage 1 or Stage 2 Polyneuropathy
Jul 27, 2018
– European Commission Decision Expected in September –
– Recommended Summary of Product Characteristics (SmPC) Includes Data on Secondary and Exploratory Endpoints, Including Results on Cardiac Parameters –
“We are delighted with this positive opinion, and today’s recommendation
by the CHMP takes us one step closer to bringing RNAi therapeutics, an
entirely new class of innovative medicines, to patients around the
world,” said
“hATTR amyloidosis is a progressively debilitating disease that often
impacts patients and their families in the prime of their lives,” said
The CHMP positive opinion is based on the evaluation of the effects of
patisiran in patients with hATTR amyloidosis and its safety profile as
demonstrated in the APOLLO Phase 3 study. The SmPC recommended by the
CHMP includes data from APOLLO primary and secondary endpoints, as well
as exploratory cardiac endpoints. The results of the APOLLO study were
published
About APOLLO
In APOLLO, the safety and efficacy of patisiran
were evaluated in a diverse, global population of hATTR amyloidosis
patients. Patients were randomized in a 2:1 ratio to receive intravenous
patisiran (0.3 mg per kilogram of body weight) or placebo once every 3
weeks for 18 months. The study showed that patisiran improved measures
of polyneuropathy, quality of life, activities of daily living,
ambulation, nutritional status and autonomic symptoms relative to
placebo in adult patients with hATTR amyloidosis. The APOLLO study used
the modified Neuropathy Impairment Score +7 (mNIS+7) to assess motor
strength, reflexes, sensation, nerve conduction and postural blood
pressure.
- Patients treated with patisiran had a mean 6.0-point decrease (improvement) in mNIS+7 score from baseline compared to a 28.0-point mean increase (worsening) for patients in the placebo group, resulting in a 34.0-point mean difference relative to placebo, after 18 months of treatment.
- While nearly all patisiran-treated patients experienced a treatment benefit relative to placebo, 56 percent of patisiran-treated patients experienced significant improvement in measures of their polyneuropathy (as assessed by mNIS+7 score) relative to their own baseline with 18 months of treatment, compared to four percent of patients who received placebo.
- As measured by the Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) Score, 51 percent of patients treated with patisiran experienced improvement in quality of life at 18 months relative to their own baseline, compared to 10 percent of the placebo-treated patients.
- Over 18 months of treatment, patients treated with patisiran experienced significant benefit vs. placebo for all other efficacy endpoints including measures of activities of daily living, walking ability, nutritional status, and autonomic symptoms.
- Patisiran was associated with favorable effects on exploratory endpoints related to cardiac structure and function in patients with cardiac involvement.
- The incidence and severity of adverse events were similar in patients receiving patisiran and placebo. The most common adverse events that occurred more frequently with patisiran than with placebo were peripheral edema and infusion-related reactions.
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.
About hATTR Amyloidosis
Hereditary transthyretin
(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. In
About RNAi
RNAi (RNA interference) is a natural cellular
process of gene silencing that represents one of the most promising and
rapidly advancing frontiers 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 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 data supporting the CHMP positive opinion, recommended SmPC, and
ongoing regulatory reviews of patisiran, the potential implications of
such data for patients, the commercial readiness of Alnylam to launch
patisiran in
None of Alnylam’s investigational therapeutics have been approved by the
View source version on businesswire.com: https://www.businesswire.com/news/home/20180727005212/en/
Source:
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom,
+1-617-682-4340
(Investors and Media)
or
Josh Brodsky,
+1-617-551-8276
(Investors)
or
Fiona McMillan, +44 1628
244960
(Media, Europe)
For Media Inquiries, please contact:
Christine Lindenboom
Chief Corporate Communications Officer media@alnylam.com 617-682-4340
For Investor Inquiries, please contact:
Josh Brodsky
VP, Investor Relations & Corporate Communications investors@alnylam.com 617-551-8276
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