Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, and taiba Middle East, a leading rare disease
company based in the United Arab Emirates and covering the Middle
East region, announced today that they have formed a Distribution
Agreement for both ONPATTRO® and GIVLAARI®, the first-ever
commercialized RNAi therapeutics, as well as another late-stage
therapy in development for Primary Hyperoxaluria Type 1.
“The cooperation with taiba allows us to address the unmet
medical needs of patients with rare diseases, particularly where
those diseases have a higher regional prevalence. That is why it is
so important that patients in the Middle East are not disadvantaged
when it comes to accessing effective treatments,” said Brendan
Martin, Vice President and Acting Head of Europe, Middle East &
Africa, and Canada, Alnylam Pharmaceuticals. “taiba has a regional
presence with operations in multiple Gulf states, and a proven
track record of marketing and distributing medicines for rare
diseases. Our collaboration marks an important first step in
bringing our current and future therapies to those patients with
urgent medical needs.”
“We are proud to collaborate with Alnylam in the Gulf states,”
said Saif Al Hasani CEO of the taiba Group. “Alnylam’s portfolio of
ground-breaking medicines will enhance our rare disease portfolio
and enable us to offer innovative treatments to patients. Those
patients with hATTR amyloidosis and other rare diseases deserve to
have the earliest possible access to novel new treatments. We look
forward to making this a reality, beginning with ONPATTRO and
GIVLAARI.”
The Agreement between Alnylam and taiba will initially cover the
Gulf states, including the Kingdom of Saudi Arabia, Kuwait,
Bahrain, Qatar, Oman and the United Arab Emirates. It includes
ONPATTRO, approved in the European Union (EU) in August 2018 for
the treatment of hATTR amyloidosis in adults with stage 1 or stage
2 polyneuropathy; GIVLAARI, approved in the EU in March 2020 for
the treatment of acute hepatic porphyria (AHP); and lumasiran, a
late-stage investigational RNAi therapeutic for the treatment of
Primary Hyperoxaluria Type 1 (PH1).
About ONPATTRO® (patisiran) ONPATTRO is an RNAi
therapeutic that was approved in the United States and Canada for
the treatment of the polyneuropathy of hATTR amyloidosis in adults.
ONPATTRO is also approved in the European Union, Switzerland and
Brazil for the treatment of hATTR amyloidosis in adults with Stage
1 or Stage 2 polyneuropathy, and in Japan for the treatment of
hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously
administered RNAi therapeutic targeting transthyretin (TTR). It is
designed to target and silence TTR messenger RNA, thereby blocking
the production of TTR protein before it is made. ONPATTRO blocks
the production of TTR in the liver, reducing its accumulation in
the body’s tissues in order to halt or slow down the progression of
the polyneuropathy associated with the disease.
Important Safety Information (ISI) for ONPATTRO®
Infusion-Related Reactions Infusion-related reactions
(IRRs) have been observed in patients treated with patisiran. In a
controlled clinical study, 19% of patisiran-treated patients
experienced IRRs, compared to 9% of placebo-treated patients. The
most common symptoms of IRRs with patisiran were flushing, back
pain, nausea, abdominal pain, dyspnoea, and headache. Hypotension
may also occur and syncope has been reported in the post-marketing
setting.
To reduce the risk of IRRs, patients should receive
premedication with a corticosteroid, paracetamol, and
antihistamines (H1 and H2 blockers) at least 60 minutes prior to
patisiran 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 Patisiran treatment leads to a decrease in
serum vitamin A levels. Patients receiving patisiran should take
oral supplementation of approximately 2500 IU vitamin A per day to
reduce the potential risk of ocular toxicity due to vitamin A
deficiency. Doses higher than 2500 IU vitamin A per day should not
be given to try to achieve normal serum vitamin A levels during
treatment with patisiran, 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. including reduced night vision or night
blindness, persistent dry eyes, eye inflammation, corneal
inflammation or ulceration, corneal thickening or corneal
perforation).
Adverse Reactions
The most common adverse reactions that occurred in patients
treated with patisiran were peripheral oedema (30%) and
infusion-related reactions (19%).
For additional information about ONPATTRO, please see the full
Prescribing Information.
About GIVLAARI® (givosiran) GIVLAARI is an RNAi
therapeutic targeting aminolevulinic acid synthase 1 (ALAS1)
approved for the treatment of adults with acute hepatic porphyria
(AHP) in the U.S. and for the treatment of AHP in adults and
adolescents aged 12 years and older in the EU. In the pivotal
ENVISION Phase 3 study, givosiran was shown to significantly reduce
the annualized rate of composite porphyria attacks that required
hospitalization, urgent healthcare visit or intravenous hemin
administration at home compared to placebo. GIVLAARI is Alnylam’s
first commercially available therapeutic based on its Enhanced
Stabilization Chemistry ESC-GalNAc conjugate technology to increase
potency and durability. GIVLAARI is administered via subcutaneous
injection once monthly at a dose based on actual body weight and
should be administered by a healthcare professional. GIVLAARI works
by specifically reducing elevated levels of ALAS1 messenger RNA,
leading to reduction of toxins associated with attacks and other
disease manifestations of AHP.
Important Safety Information for GIVLAARI®
Contraindications Givosiran is contraindicated in
patients with known severe hypersensitivity to givosiran. Reactions
have included anaphylaxis.
Anaphylactic Reaction Anaphylaxis has occurred with
givosiran treatment (<1% of patients in clinical trials). Ensure
that medical support is available to appropriately manage
anaphylactic reactions when administering givosiran. Monitor for
signs and symptoms of anaphylaxis. If anaphylaxis occurs,
immediately discontinue administration of GIVOSIRAN and institute
appropriate medical treatment.
Hepatic Toxicity Transaminase elevations (ALT) of at
least 3 times the upper limit of normal (ULN) were observed in 15%
of patients receiving givosiran in the placebo-controlled trial.
Transaminase elevations primarily occurred between 3 to 5 months
following initiation of treatment.
Measure liver function tests prior to initiating treatment with
givosiran, repeat every month during the first 6 months of
treatment, and as clinically indicated thereafter. Interrupt or
discontinue treatment with givosiran for severe or clinically
significant transaminase elevations. In patients who have dose
interruption and subsequent improvement, reduce the dose to 1.25
mg/kg once monthly. The dose may be increased to the recommended
dose of 2.5 mg/kg once monthly if there is no recurrence of severe
or clinically significant transaminase elevations at the 1.25 mg/kg
dose.
Renal Toxicity Increases in serum creatinine levels and
decreases in estimated glomerular filtration rate (eGFR) have been
reported during treatment with givosiran. In the placebo-controlled
study, 15% of patients receiving givosiran experienced a
renally-related adverse reaction. The median increase in creatinine
at Month 3 was 0.07 mg/dL. Monitor renal function during treatment
with givosiran as clinically indicated.
Injection Site Reactions In placebo-controlled and
open-label clinical studies, injection site reactions have been
reported in 36 % of patients Symptoms included erythema, pain,
pruritus, rash, discoloration, or swelling around the injection
site. Three patients (2.7 %) experienced a single, transient,
recall reaction of erythema at a prior injection site with a
subsequent dose administration.
Drug Interactions Caution is recommended during the use
of medicinal products that are substrates of CYP1A2 or CYP2D6 while
on treatment with Givosiran as this medicinal product may increase
or prolong their therapeutic effect, or alter their adverse event
profiles Consider decreasing the CYP1A2 or CYP2D6 substrate dosage
in accordance with the approved product labelling.
Adverse Reactions The most common adverse reactions that
occurred in patients receiving givosiran were nausea (32%),
injection site reactions (36 %) and fatigue (22.5 %). The adverse
reactions resulting in discontinuation of treatment were elevated
transaminases (0.9 %) and anaphylactic reaction (0.9 %).
For additional information about GIVLAARI, please see full
Prescribing Information.
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 new class of medicines, known as RNAi
therapeutics, is now a reality. 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 or disease pathway 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 Pharmaceuticals 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, hepatic infectious, and central nervous system
(CNS)/ocular 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 RNAi
therapeutics platform. Alnylam’s commercial RNAi therapeutic
products are ONPATTRO® (patisiran), approved in the U.S., EU,
Canada, Japan, Brazil, and Switzerland, and GIVLAARI® (givosiran),
approved in the U.S and the EU. Alnylam has a deep pipeline of
investigational medicines, including six product candidates that
are in late-stage development. Alnylam is executing 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 is headquartered in
Cambridge, MA.
About taiba taiba is a leading specialty marketing, sales
and distribution company in the MENA region, providing innovative
treatments to patients suffering from orphan and rare diseases.
taiba’s vision is to cover the unmet medical need in its region,
providing high quality products and a high level of service to
healthcare organizations and hospitals, with a commitment to
patient treatment. taiba’s focus is addressing the needs of rare
disease patients and providing them with access to innovative
medicines, either through named patient sales or commercialization.
Over the last ten years, taiba has earned a reputation as a leading
regional company in its segment and currently represents leading
multinational companies that are pioneers in their fields such as
Alexion, Aegerion, Biomarin, Chiesi, Dyax, Genzyme, Lucane, Veloxis
and Vertex.
Alnylam Forward Looking Statements Various statements in
this release concerning Alnylam's future expectations, plans and
prospects, including, without limitation, Alnylam’s expectations
regarding its collaboration with taiba and the possibility to
address the unmet medical needs of patients with rare diseases,
particularly where those diseases have a higher regional
prevalence, its views with respect to taiba’s track record of
marketing and distributing medicines for rare diseases, and
expectations regarding the continued execution on 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: the direct or
indirect impact of the COVID-19 global pandemic or a future
pandemic, such as the scope and duration of the outbreak,
government actions and restrictive measures implemented in
response, material delays in diagnoses of rare diseases, initiation
or continuation of treatment for diseases addressed by Alnylam
products, or in patient enrollment in clinical trials, potential
supply chain disruptions, and other potential impacts to Alnylam’s
business, the effectiveness or timeliness of steps taken by Alnylam
to mitigate the impact of the pandemic, and Alnylam’s ability to
execute business continuity plans to address disruptions caused by
the COVID-19 or a future pandemic; Alnylam's ability to discover
and develop novel drug candidates and delivery approaches and
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, including lumasiran, or its marketed products;
obtaining, maintaining and protecting intellectual property;
intellectual property matters including potential patent litigation
relating to its platform, products or product candidates; obtaining
regulatory approval for its product candidates, including
lumasiran, and maintaining regulatory approval and obtaining
pricing and reimbursement for its products, including ONPATTRO and
GIVLAARI; progress in continuing to establish a commercial and
ex-United States infrastructure; successfully launching, marketing
and selling its approved products globally, including ONPATTRO and
GIVLAARI, and achieving net product revenues for ONPATTRO within
its revised expected range during 2020; Alnylam’s ability to
successfully expand the indication for ONPATTRO in the future;
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 within the ranges of
guidance provided by Alnylam through the implementation of further
discipline in operations to moderate spend and its ability to
achieve a self-sustainable financial profile in the future without
the need for future equity financing; Alnylam’s ability to
establish and maintain strategic business alliances and new
business initiatives, including completing an agreement for funding
by Blackstone of certain R&D activities for vutrisiran and
ALN-AGT; Alnylam's dependence on third parties, including
Regeneron, for development, manufacture and distribution of certain
products, including eye and CNS products, Ironwood, for assistance
with the education about and promotion of GIVLAARI, and Vir for the
development of ALN-COV and other potential RNAi therapeutics
targeting SARS-CoV-2 and host factors for SARS-CoV-2; 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20200708005495/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media)
617-682-4340
Fiona McMillan (Media, Europe) +44 1628 244960
Taiba Group Jobin Korah (Media, taiba Middle East)
taibame@taibahealthcare.com + 9714-3752750
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