– First and only FDA-approved
subcutaneous treatment option for anti-aquaporin-4 antibody
positive NMOSD that can be self-administered by a person with NMOSD
or a caregiver every four weeks –
– First and only approved therapy for NMOSD
designed to target and inhibit interleukin-6 receptor activity,
using novel recycling antibody technology –
– Approval supported by one of the largest
clinical trial programs undertaken for this rare disease –
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX:
RHHBY), today announced that the U.S. Food and Drug Administration
(FDA) has approved Enspryng™ (satralizumab-mwge) as the first and
only subcutaneous treatment for adults living with anti-aquaporin-4
(AQP4) antibody positive neuromyelitis optica spectrum disorder
(NMOSD). NMOSD is a rare, lifelong and debilitating autoimmune
disorder of the central nervous system, often misdiagnosed as
multiple sclerosis, that primarily damages the optic nerve(s) and
spinal cord, causing blindness, muscle weakness and paralysis.
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“Today’s FDA approval of Enspryng, the first subcutaneous NMOSD
treatment using novel recycling antibody technology, builds upon
the work we’ve done in multiple sclerosis with Ocrevus to develop
first-in-class medicines and further the scientific understanding
of neuroimmunological diseases,” said Levi Garraway, M.D., Ph.D.,
chief medical officer and head of Global Product Development. “We
thank the NMOSD community, including patients and investigators who
participated in Enspryng clinical trials.”
Enspryng is a humanized monoclonal antibody and the only
approved therapy for NMOSD designed to target and inhibit
interleukin-6 (IL-6) receptor activity, believed to play a key role
in the inflammation associated with NMOSD. The treatment was
designed using novel recycling antibody technology, which, compared
to conventional technology, allows for longer duration of antibody
circulation and subcutaneous dosing every four weeks.
“For people with NMOSD, relapses can cause devastating,
irreversible and disabling neurological effects,” said Professor
Jeffrey Bennett, University of Colorado Neurology &
Ophthalmology, and investigator for the Enspryng pivotal clinical
trials. “Having an approved therapy that can be administered
subcutaneously in the home and has demonstrated an impact on the
frequency of relapses is an important advancement for
patients.”
“We are very optimistic the addition of this new approved
treatment option will make a meaningful difference for those living
with NMOSD, those who love and support them and the doctors who
treat them,” said Victoria Jackson, founder, The Guthy-Jackson
Charitable Foundation. “When my daughter was diagnosed with NMOSD
in 2008, there were no approved treatment options, and a critical
lack of resources and understanding for people living with this
disabling disorder. After years of dedicated effort and
collaboration, the FDA approval of Enspryng exemplifies how
patients, industry, and academia can find solutions together.”
Enspryng can be administered in the home by a person living with
NMOSD or a caregiver following training from a healthcare provider.
Enspryng treatment is administered every four weeks after an
initial loading dose.
Enspryng will be available in the United States in two weeks.
Genentech is committed to helping patients access the medicines
prescribed by their physician. For people with NMOSD, the Enspryng
Access Solutions team is available to answer questions, provide
product education, injection training and help families understand
insurance coverage and navigate appropriate financial assistance
options to start and stay on Enspryng. Patients can call
1-844-NSPRYNG (844-677-7964) to speak to a Patient Navigator or
visit http://www.Enspryng.com.
FDA approval is based on results from one of the largest
pivotal clinical trial programs undertaken for this rare
neurological disorder
This approval is supported by results from two randomized
controlled Phase III clinical trials, the SAkuraStar and SAkuraSky
studies, in which Enspryng demonstrated robust and sustained
efficacy and a favorable safety profile in adults with AQP4
antibody positive NMOSD. Results were sustained for 96 weeks,
significantly reducing the risk of relapse compared with placebo as
a monotherapy and when used concurrently with baseline
immunosuppressant therapy (IST), which has commonly been used to
manage NMOSD symptoms associated with relapses.
In the SAkuraStar monotherapy study’s AQP4 antibody positive
subgroup, 76.5% of Enspryng-treated patients were relapse-free at
96 weeks, compared to 41.1% with placebo. In the SAkuraSky study,
which evaluated Enspryng when used concurrently with baseline IST,
91.1% of Enspryng-treated AQP4 antibody positive subgroup patients
were relapse-free at 96 weeks, compared to 56.8% with placebo. The
primary endpoint of both SAkuraStar and SAkuraSky was time to first
protocol-defined relapse (PDR) adjudicated by an independent review
committee in the double-blind period.
The most common adverse reactions with Enspryng (incidence ≥
15%) were nasopharyngitis, headache, upper respiratory tract
infection, gastritis, rash, arthralgia, extremity pain, fatigue and
nausea.
About SAkuraStar and SAkuraSky in NMOSD
SAkuraStar is a Phase III multicenter, randomized, double-blind,
placebo-controlled study to evaluate the efficacy and safety of
Enspryng monotherapy administered to patients with NMOSD. The
primary endpoint is the time to first protocol-defined relapse
(PDR), adjudicated by an independent review committee in the
double-blind period. Results from the SAkuraStar study were
presented at the 35th Congress of the European Committee for
Treatment and Research in Multiple Sclerosis (ECTRIMS), September
11-13, 2019, and were published in the May 1, 2020 edition of The
Lancet Neurology.
Ninety-five adult patients were randomized to either of the
following two treatment groups in a 2:1 ratio: Enspryng (120 mg) or
placebo. Both treatments were administered subcutaneously at week
0, 2, and 4. The subsequent treatment was continued at 4-week
intervals. The double-blind treatment period ended at 1.5 years
after the enrollment of the last patient. After experiencing a PDR
or upon completion of the study, patients in both groups were
offered treatment with Enspryng in an open label extension (OLE)
period. Patients with aquaporin-4 (AQP4) antibody positive or
negative neuromyelitis optica (NMO, as defined by the diagnostic
criteria in 2006) and those with AQP4 antibody positive NMOSD were
enrolled. The number of AQP4 antibody negative patients was limited
to approximately 33% of the total population of the study. Data
have shown that AQP4 antibody positive patients may experience a
greater likelihood of relapse and poorer long-term outcomes than
AQP4 antibody negative patients.
SAkuraSky is a Phase III multicenter, randomized, double-blind,
placebo-controlled study to evaluate the efficacy and safety of
Enspryng added to baseline immunosuppressant therapy in patients
with NMOSD. The primary endpoint was the time to first PDR as
adjudicated by an independent review committee in the double-blind
period. Results from the SAkuraSky study were published in the
November 28, 2019 edition of the New England Journal of Medicine
(NEJM).
Seventy-six adult patients were randomized to either of the
following two treatment groups in a 1:1 ratio: Enspryng (120 mg) or
placebo added to baseline therapy (azathioprine, mycophenolate
mofetil and/or corticosteroids). Both treatments were administered
subcutaneously at week 0, 2, and 4. The subsequent treatment was
continued at 4-week intervals. The double-blind treatment period
ended when patients experienced a PDR; the study ended when the
total number of PDRs reached 26. After experiencing a PDR or upon
completion of the study, patients in both groups were offered
treatment with Enspryng in an OLE period. Patients with AQP4
antibody positive or negative neuromyelitis optica (NMO, as defined
by diagnostic criteria in 2006) and those with AQP4 antibody
positive NMOSD were enrolled. AQP4 antibody negative patients
represented approximately 30% of the SAkuraSky study
population.
About neuromyelitis optica spectrum disorder (NMOSD)
NMOSD is a rare, lifelong and debilitating autoimmune condition
of the central nervous system that primarily damages the optic
nerve(s) and spinal cord, causing blindness, muscle weakness and
paralysis. People with NMOSD experience unpredictable, severe
relapses directly causing cumulative, permanent, neurological
damage and disability. In some cases, relapse can result in death.
NMOSD affects over 10,000 people in Europe, up to 15,000 people in
the United States and approximately 200,000 people worldwide. NMOSD
can affect individuals of any age, race and gender, but is most
common among women in their 30s and 40s, and appears to occur at
higher rates in people of African or Asian background. There is
some evidence that people of African or Asian descent may also
experience a more severe disease course.
NMOSD is commonly associated with pathogenic antibodies (AQP4)
that target and damage a specific cell type, called astrocytes,
resulting in inflammatory lesions of the optic nerve(s), spinal
cord and brain. AQP4 antibodies are detectable in the blood serum
of around 70-80% of NMOSD patients.
Although most cases of NMOSD can be confirmed through diagnostic
tests, people living with the condition are still frequently
misdiagnosed with multiple sclerosis. This is due to overlapping
characteristics of the two disorders, including a higher prevalence
in women, similar symptoms and the fact that both are relapse-based
conditions.
About Enspryng™ (satralizumab-mwge)
Enspryng, which was designed by Chugai, a member of the Roche
group, is a humanized monoclonal antibody that targets IL-6
receptor activity. The cytokine IL-6 is believed to be a key driver
in NMOSD, triggering the inflammation cascade and leading to damage
and disability. Enspryng was designed using novel recycling
antibody technology, which compared to conventional technology,
allows for longer duration of the antibody and subcutaneous dosing
every four weeks.
Positive Phase III results for Enspryng, as both monotherapy and
used concurrently with baseline immunosuppressant therapy, suggest
that IL-6 inhibition is an effective therapeutic approach for
NMOSD. The Phase III clinical development program for Enspryng
includes two studies: SAkuraStar and SAkuraSky.
Enspryng is also approved in Canada, Japan and Switzerland.
Applications are under review with numerous regulators, including
in the European Union and China.
Enspryng has been designated as an orphan drug in the United
States, Europe and Japan. In addition, it was granted Breakthrough
Therapy Designation for the treatment of NMOSD by the FDA in
December 2018.
What is Enspryng?
Enspryng is a prescription medicine used to treat neuromyelitis
optica spectrum disorder (NMOSD) in adults who are aquaporin-4
(AQP4) antibody positive.
It is not known if Enspryng is safe and effective in
children.
Important Safety Information
Patients should not take Enspryng if they:
- are allergic to satralizumab-mwge or any of the ingredients in
Enspryng
- have an active hepatitis B infection
- have active or untreated inactive (latent) tuberculosis
(TB)
Enspryng may cause serious side effects including:
- Infections. Enspryng can increase risk of serious
infections some of which can be life-threatening. Patients should
speak with their healthcare provider if they are being treated for
an infection and call right away if there are signs of an
infection, with or without a fever, such as:
- chills, feeling tired, muscle aches, cough that will not go
away or a sore throat
- skin redness, swelling, tenderness, pain or sores on the
body
- diarrhea, belly pain, or feeling sick
- burning when urinating or urinating more often than usual
- A healthcare provider will check for infection and treat it if
needed before starting or continuing to take Enspryng
- A healthcare provider should test for hepatitis and TB before
initiating Enspryng
- All required vaccinations should be completed before starting
Enspryng. People using Enspryng should not be given ‘live’ or
‘live-attenuated’ vaccines. ‘Live’ or ‘live-attenuated’ vaccines
should be given at least 4 weeks before a patient starts Enspryng.
A healthcare provider may recommend that a patient receive a
‘non-live’ (inactivated) vaccine, such as some of the seasonal flu
vaccines. If a patient plans to get a ‘non-live’ (inactivated)
vaccine it should be given, whenever possible, at least 2 weeks
before starting Enspryng
- Increased liver enzymes. A healthcare provider should
order blood tests to check patient liver enzymes before and while
taking Enspryng. A healthcare provider will dictate how often these
blood tests are needed. Patients should complete all follow-up
blood tests as ordered by a healthcare provider. A healthcare
provider may wait to start Enspryng if liver enzymes are
increased
- Low neutrophil count. Enspryng can cause a decrease in
neutrophil counts in the blood. Neutrophils are white blood cells
that help the body fight off bacterial infections. A healthcare
provider should order blood tests to check neutrophil counts while
a patient is taking Enspryng.
- Serious allergic reactions that may be life-threatening
have happened with other medicines like Enspryng. Patients should
call their healthcare provider right away if they have any of these
symptoms of an allergic reaction:
- shortness of breath or trouble breathing
- swelling of lips, face, or tongue
- dizziness or feeling faint
- moderate or severe stomach (abdominal) pain or vomiting
- chest pain
Before taking Enspryng, patients should tell their healthcare
provider about all of their medical conditions, including if
they
- have or think they have an infection
- have liver problems
- have ever had hepatitis B or are a carrier of the hepatitis B
virus
- have had or have been in contact with someone with TB
- have had a recent vaccination or are scheduled to receive any
vaccination
- are pregnant, think they might be pregnant, or plan to become
pregnant. It is not known if Enspryng will harm one’s unborn
baby
- are breastfeeding or plan to breastfeed. It is not known if
Enspryng passes into breast milk. Patients should speak with their
healthcare provider about the best way to feed one’s baby while on
treatment with Enspryng
Patients should tell their healthcare provider about all the
medicines they take, including prescription and
over-the-counter medicines, vitamins and herbal supplements
The most common side effects of Enspryng include:
- sore throat, runny nose (nasopharyngitis)
- headache
- upper respiratory tract infection
- rash
- fatigue
- nausea
- extremity pain
- inflammation of the stomach lining
- joint pain
For more information about the risks and benefit profiles of
Enspryng, patients should ask their healthcare provider.
Patients may report side effects to the FDA at 1-800-FDA-1088 or
http://www.fda.gov/medwatch. Patients may also report side effects
to Genentech at 1-888-835-2555.
Please see the full Prescribing Information for additional
Important Safety Information.
About Genentech in neuroscience
Neuroscience is a major focus of research and development at
Genentech and Roche. Our goal is to pursue groundbreaking science
to develop new treatments that help improve the lives of people
with chronic and potentially devastating diseases.
Genentech and Roche are investigating more than a dozen
medicines for neurological disorders, including multiple sclerosis,
neuromyelitis optica spectrum disorder, Alzheimer’s disease,
Huntington’s disease, Parkinson’s disease, Duchenne muscular
dystrophy and autism spectrum disorder. Together with our partners,
we are committed to pushing the boundaries of scientific
understanding to solve some of the most difficult challenges in
neuroscience today.
About Genentech
Founded more than 40 years ago, Genentech is a leading
biotechnology company that discovers, develops, manufactures and
commercializes medicines to treat patients with serious and
life-threatening medical conditions. The company, a member of the
Roche Group, has headquarters in South San Francisco, California.
For additional information about the company, please visit
http://www.gene.com.
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