- Expedited Access to OXLUMO Aims to Support
Children and Adults Living with PH1 who Face Inevitable Disease
Progression and Irreversible Kidney Damage in the Absence of New
Treatment Options -
- New Value-Based Agreement Framework Includes
an Innovative Patient Need Adjustment that Offers Payers Increased
Cost Predictability Across the Entire Spectrum of PH1 Patient Ages,
from Infant to Adult -
- Express Scripts, Harvard Pilgrim, and
Highmark are Among Leading Payers Pursuing Agreements in Principle
-
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today a new framework for
value-based agreements (VBAs) designed to help people with primary
hyperoxaluria type 1 (PH1) gain access to OXLUMO™ (lumasiran). Now
approved by the U.S. Food and Drug Administration (FDA) for the
treatment of PH1 to lower urinary oxalate levels in pediatric and
adult patients, OXLUMO is the first-ever approved targeted
therapeutic that substantially curbs oxalate production in patients
living with PH1, an ultra-rare genetic disease characterized by
oxalate overproduction.
Alnylam is in active discussions with leading payers and has
reached an agreement in principle with Express Scripts, Harvard
Pilgrim, and Highmark to pursue VBAs for OXLUMO.
Oxalate overproduction in PH1 results in elevated urinary
oxalate and the deposition of calcium oxalate crystals in the
kidneys and urinary tract. People with PH1 typically endure
intensive management of debilitating, painful and recurrent kidney
stones. With limited treatment options previously available, the
disease would inevitably progress to kidney failure,
nephrocalcinosis (calcification of the kidneys), and multi-organ
dysfunction as a consequence of systemic oxalosis (the spread of
oxalate to organs and tissues outside of the kidneys).1 People with
PH1 often present with kidney failure at the time of diagnosis.2
PH1 patients with renal failure undergo dialysis almost daily, for
up to 10-12 hours per day and night.2 A dual or sequential
liver/kidney transplant is then typically performed to resolve the
underlying metabolic defect in the liver and restore kidney
function,2 but these interventions are associated with life-long
immunosuppression and a high risk of morbidity and mortality. Until
now, there have been no FDA-approved pharmaceutical therapies for
PH1. There are approximately one to three individuals per million
across the U.S. and Europe with a confirmed PH1 diagnosis, of those
it is estimated that 1,000 - 1,700 individuals have not yet
received a liver transplant, making them potentially eligible for
treatment.3
VBA Framework for OXLUMO
Building on the Alnylam Patient Access Philosophy and Alnylam’s
ongoing commitment to deliver fair value to payers and providers,
the Company has worked with payers on a new and enhanced VBA
framework. Since OXLUMO is indicated for both pediatric and adult
patients, and is dosed by weight, related costs can vary relative
to each patient and use over time. As such, Alnylam has structured
a new VBA component that specifically addresses many payers’
concerns for budget predictability and value, particularly for
ultra-rare orphan disease therapies that are administered across a
wide spectrum of ages from infants to adults.
The new VBA component, called a Patient Need Adjustment (PNA),
is now being added to Alnylam’s overall VBA offering for OXLUMO.
Participating payers will qualify for the PNA rebate if the average
number of vials utilized by a plan member exceeds an established
threshold, providing payers with greater short-term and long-term
predictability. The PNA was designed to mitigate the risk of
escalating or varying costs associated with dosing requirements,
thereby accelerating access for people diagnosed with PH1.
To further address budget predictability, Alnylam is also making
available its Prevalence Based Adjustment (PBA) component, which
was first introduced last year for Alnylam’s second-approved
therapy, GIVLAARI® (givosiran). There are often uncertainties in
diagnosis rates and disease prevalence estimates in ultra-rare,
poorly diagnosed orphan diseases, making it challenging for payers
to predict the number of patients who will be covered within their
plans. This feature triggers a rebate to participating payers if
the number of diagnosed patients they cover exceeds current
epidemiologic estimates for PH1.
“Value-based agreements for OXLUMO focus on three critical
priorities for Alnylam in PH1: first, do everything possible to
speed covered access to OXLUMO to begin reducing oxalate
overproduction in patients; second, link OXLUMO price to actual
value delivered; and, third, remove the cost variability to payers
associated with medicines administered across a broad age range,
while also addressing budget unpredictability concerns arising from
uncertain prevalence estimates in ultra-rare diseases,” said Andy
Orth, Senior Vice President, Head of U.S. Region at Alnylam. “Our
new Patient Need Adjustment component was born out of direct
feedback from our payer partners, and experience with our first two
approved therapies. We’ve listened to the needs of payers covering
PH1 patients and have responded with what we believe is an
attractive solution. At Alnylam, we will continue to pursue
commercial innovation that offers good value and straightforward
pricing for potentially transformative RNAi therapies like OXLUMO,
the first-ever approved medicine for PH1.”
“We’re pleased to expand on our legacy of innovative contracting
with Alnylam and support access to a transformative therapy for
patients with primary hyperoxaluria type 1. The Patient Need
Adjustment is an innovative component of the OXLUMO value-based
agreement intended to provide budget predictability,” said Michael
Sherman, M.D., M.B.A., Chief Medical Officer of Harvard Pilgrim
Health Care.
“With an expanding number of medicines being approved for rare
and ultra-rare diseases, payers and plans face the increasingly
difficult task of predicting costs for therapies while establishing
coverage for patients in need. The Patient Need Adjustment is a
responsible and very welcome model to address certain payer
challenges, creating a solution that mitigates cost risks
associated with ultra-rare medicines administered across a broad
range of patient age groups,” said Steve Miller, M.D., Chief
Medical Officer, CIGNA, Express Scripts.
“Highmark is focused on ensuring that our members have access to
effective therapies, including increasingly available rare and
ultra-rare disease therapies, while addressing costs,” said Sean
Quinn, PharmD., Pharmaceutical Manufacturer Relations Director for
Highmark Inc. “We have emerged as a leader in the value-based
agreements space for therapies that address a number of chronic,
high-cost health conditions. VBAs can help speed access to new
medicines by tying outcomes to clinical trial performance, but
payers are seeking other barometers for predicting need, which can
be complex for therapies indicated across a spectrum of patient
ages. We have collaborated with Alnylam to tackle new and evolving
problems in rare and ultra-rare disease drug coverage, and are
proud to work on this innovative Patient Need Adjustment to help
people seeking treatment with OXLUMO.”
Alnylam’s Patient Access Philosophy
The innovative agreements announced today further reinforce
Alnylam’s Patient Access Philosophy, which was created three years
ago to seek solutions for patients, deliver value to payers and
physicians, and remove barriers to access. This Philosophy commits
that Alnylam will not increase the price of OXLUMO by more than the
consumer price index for urban consumers (CPI-U), a measure of
inflation, unless it is approved for new conditions by the U.S.
FDA. Patients with private (commercial) insurance are expected to
have little-to-no out-of-pocket copayments for OXLUMO. To see
Alnylam’s progress, visit
https://www.alnylam.com/about-alnylam/patient-access-philosophy/.
Alnylam Assist® and Alnylam Act®
Alnylam offers multiple programs to support patients. A
comprehensive patient support services program, Alnylam Assist®,
will help patients gain access to OXLUMO. Alnylam Assist® will
offer an in-house team of Case Managers to assist patients with
verification of insurance benefits and financial assistance for
those who qualify. Patients will also be eligible to receive
support from Patient Education Liaisons, who can answer questions
about disease and treatment. Physicians and patients can learn more
about Alnylam’s comprehensive patient services by visiting
AlnylamAssist.com or calling 1-833-256-2748. Alnylam also continues
to offer its third-party genetic testing service in the U.S.,
Canada, and Brazil, called Alnylam Act®. The program is provided at
no charge to patients and their physicians and aims to reduce the
time to accurate diagnoses for genetic diseases, such as PH1.
Visit OXLUMO.com for more information, including full
Prescribing Information.
IMPORTANT SAFETY INFORMATION
Adverse Reactions
The most common adverse reaction that occurred in patients
treated with OXLUMO was injection site reaction (38%). Symptoms
included erythema, pain, pruritus, and swelling.
Pregnancy and Lactation
No data are available on the use of OXLUMO in pregnant women. No
data are available on the presence of OXLUMO in human milk or its
effects on breastfed infants or milk production. Consider the
developmental and health benefits of breastfeeding along with the
mother’s clinical need for OXLUMO and any potential adverse effects
on the breastfed child from OXLUMO or the underlying maternal
condition.
For additional information about OXLUMO, please see the full
Prescribing Information.
About OXLUMO™ (lumasiran)
OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1
(HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) to
lower urinary oxalate levels in pediatric and adult patients. HAO1
encodes glycolate oxidase (GO), an enzyme upstream of the
disease-causing defect in PH1. OXLUMO works by degrading HAO1
messenger RNA and reducing the synthesis of GO, which inhibits
hepatic production of oxalate – the toxic metabolite responsible
for the clinical manifestations of PH1. In the pivotal ILLUMINATE-A
study, OXLUMO was shown to significantly reduce levels of urinary
oxalate relative to placebo, with the majority of patients reaching
normal or near-normal levels. Injection site reactions (ISRs) were
the most common drug-related adverse reaction. In the ILLUMINATE-B
pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety
profile consistent to that observed in ILLUMINATE-A. OXLUMO
utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc
conjugate technology designed to increase potency and durability.
OXLUMO is administered via subcutaneous injection once monthly for
three months, then once quarterly thereafter at a dose based on
actual body weight. For patients who weigh less than 10 kg, ongoing
dosing remains monthly. OXLUMO should be administered by a
healthcare professional. For more information about OXLUMO, visit
OXLUMO.com.
About Primary Hyperoxaluria Type 1 (PH1)
PH1 is an ultra-rare genetic disease that affects an estimated
one to three individuals per million in the United States and
Europe. PH1 is characterized by oxalate overproduction in the
liver. The excess oxalate results in the deposition of calcium
oxalate crystals in the kidneys and urinary tract and can lead to
the formation of painful and recurrent kidney stones and
nephrocalcinosis. Renal damage is caused by a combination of
tubular toxicity from oxalate, calcium oxalate deposition in the
kidneys, and urinary obstruction by calcium oxalate stones. PH1 is
associated with a progressive decline in kidney function, which
exacerbates the disease as the excess oxalate can no longer be
effectively excreted, resulting in subsequent accumulation and
deposition of oxalate in bones, eyes, skin, and heart, leading to
severe illness and death. Management options to date were limited
to hyperhydration, crystallization inhibitors and, in a minority of
patients with a specific genotype, pyridoxine (vitamin B6). These
measures do not adequately address oxalate overproduction and are
intended to delay inevitable progression to kidney failure and the
need for intensive dialysis as a bridge to a dual or sequential
liver/kidney transplant. Liver transplantation is the only
intervention that addresses the underlying metabolic defect, but is
associated with high morbidity and mortality, and life-long
immunosuppression. Until today, there were no approved
pharmaceutical therapies for PH1.
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), GIVLAARI®
(givosiran), and OXLUMO™ (lumasiran). 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. For more information about our people, science and
pipeline, please visit www.alnylam.com and engage with us on
Twitter at @Alnylam or on LinkedIn.
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 safety and efficacy of OXLUMO
as demonstrated in the ILLUMINATE-A and ILLUMINATE-B Phase 3
studies and the potential for OXLUMO to address the underlying
pathophysiology of PH1 in adults, children and infants, the
potential for OXLUMO to substantially curb or reduce oxalate
production in pediatric and adult patients with PH1, expectations
regarding Alnylam’s new framework for VBAs designed to help people
with PH1 gain covered access to OXLUMO, link OXLUMO price to actual
value delivered, remove the cost variability to payers associated
with medicines administered across a broad age range, and address
budget unpredictability concerns arising from uncertain estimated
prevalence of ultra-rare diseases such as PH1, expectations
regarding the attractiveness of Alnylam’s new VBA component to
payers, expectations regarding the entry into definitive VBA
agreements for OXLUMO with leading payers, including Express
Scripts, Harvard Pilgrim, and Highmark, among others, and
expectations regarding the potential for Alnylam to meet or exceed
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 any 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 any 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 or its other marketed products, including
OXLUMO; 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, and maintaining regulatory approval and obtaining
pricing and reimbursement for its products, including ONPATTRO,
GIVLAARI and OXLUMO; progress in continuing to establish an
ex-United States infrastructure; successfully launching, marketing
and selling its approved products globally, including ONPATTRO,
GIVLAARI and OXLUMO, 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; Alnylam's dependence on
third parties, including Regeneron, for development, manufacture
and distribution of certain products, including eye and CNS
products, 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.
1Lorenzo V, Torres A, Salido E. Primary hyperoxaluria.
Nefrologia. 2014 May 21;34(3):398-412. English, Spanish. doi:
10.3265/Nefrologia.pre2014.Jan.12335. Epub 2014 Apr 30. PMID:
24798559.
2Cochat P, et al. Primary hyperoxaluria Type 1: indications for
screening and guidance for diagnosis and treatment. Nephrol Dial
Transplant. 2012 May;27(5):1729-36. doi: 10.1093/ndt/gfs078. PMID:
22547750.
3Lumasiran, in Development for the Treatment of Primary
Hyperoxaluria Type I. Alnylam RNAi Roundtable 2020; (August 10,
2020). Available at:
https://event.webcasts.com/viewer/event.jsp?ei=1351579&tp_key=7522004032
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Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) +1-617-682-4340
Josh Brodsky (Investors) +1-617-551-8276
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