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FDA Grants Priority Review to Genentech’s Rituxan (Rituximab) in Children with Two Rare Blood Vessel Disorders

June 12, 2019 Off By BusinessWire

There are currently no FDA approved treatments for children living
with granulomatosis with polyangiitis (GPA) or microscopic
polyangiitis (MPA)
The PePRS study is the first global trial of Rituxan in pediatric
patients with GPA or MPA
Rituxan in combination with glucocorticoids is the only
FDA-approved therapy for adults with these two rare forms of vasculitis
If approved, this would be the first pediatric indication for
Rituxan

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY),
announced today that the U.S. Food and Drug Administration (FDA) has
accepted the company’s supplemental Biologics License Application (sBLA)
and granted Priority Review for the use of Rituxan^®
(rituximab), in combination with glucocorticoids (GCC), for the
treatment of granulomatosis with polyangiitis (GPA) and microscopic
polyangiitis (MPA) in children two years of age and older. GPA and MPA
are rare, potentially life-threatening diseases affecting small and
medium sized blood vessels.¹

“We are committed to delivering new treatment options for rare diseases,
such as pediatric GPA and MPA, for which there are currently no approved
medicines,” said Sandra Horning, M.D., chief medical officer and head of
Global Product Development. “We will continue to work closely with the
FDA to bring Rituxan to children with these two serious and potentially
life-threatening diseases.”

Priority Review Designation is granted to medicines that, if approved,
the FDA has determined to have the potential to provide significant
improvements in the safety or effectiveness of the treatment of a
serious disease.

The sBLA was submitted based on data from the PePRS study, a Phase IIa,
global, open-label, single-arm study investigating the safety,
pharmacodynamics/pharmacokinetics and exploratory efficacy of
intravenous Rituxan in 25 patients with severe GPA or MPA between six
and 17 years of age.² Treatment with four weekly infusions of
Rituxan in combination with a tapering course of oral glucocorticoids
was assessed in newly diagnosed or relapsing active GPA or MPA pediatric
patients.

Rituxan is currently indicated for the treatment of four autoimmune
indications. The FDA approved Rituxan for rheumatoid arthritis (RA) in
2006, for the treatment of adults with GPA and MPA in 2011, and for
adults with pemphigus vulgaris in 2018. Since 2006, more than 900,000
patients have been treated with Rituxan for autoimmune conditions
worldwide. If approved, this would be the first pediatric indication for
Rituxan.

About Granulomatosis with Polyangiitis and Microscopic Polyangiitis

Granulomatosis with Polyangiitis (GPA) (formerly known as Wegener’s
Granulomatosis) and Microscopic Polyangiitis (MPA) are two types of
ANCA-associated vasculitis (AAV).³ AAV is a form of
vasculitis, or blood vessel inflammation, that primarily affects small
blood vessels.³ In general, GPA and MPA both affect the small
blood vessels of the kidneys, lungs, sinuses, and a variety of other
organs, but the diseases may affect each person differently.^1,4Both
GPA and MPA are considered rare diseases, with an estimated prevalence
in the United States of up to three cases per 100,000 people.^1,4
Cases of pediatric onset GPA and MPA are even more rare and are
associated with severe, potentially life-threatening symptoms.⁵

What autoimmune diseases does Rituxan treat?

Rheumatoid arthritis (RA): with another prescription medicine called
methotrexate, to reduce the signs and symptoms of moderate to severe
active RA in adults, after treatment with at least one other medicine
called a tumor necrosis factor (TNF) antagonist has been used and did
not work well enough.

Granulomatosis with Polyangiitis (GPA) (Wegener’s Granulomatosis) and
Microscopic Polyangiitis (MPA): with glucocorticoids, to treat adults
with GPA and MPA.

Pemphigus vulgaris (PV): to treat adults with moderate to severe
PV.

It is not known if Rituxan is safe or effective in children.

Important Side Effect Information

What is the most important information patients should know about
Rituxan?

Rituxan can cause serious side effects that can lead to death,
including:

Infusion-Related Reactions: Infusion-related
reactions are very common side effects of Rituxan treatment. Serious
infusion-related reactions can happen during your infusion or within
24 hours after your infusion of Rituxan. Your healthcare provider
should give you medicines before your infusion of Rituxan to decrease
your chance of having a severe infusion-related reaction
Severe Skin and Mouth Reactions: Tell
your healthcare provider or get medical help right away if you get any
of these symptoms at any time during your treatment with Rituxan:
painful sores or ulcers on your skin, lips, or in your mouth;
blisters; peeling skin; rash; or pustules
Hepatitis B Virus (HBV) Reactivation: If
you have had hepatitis B or are a carrier of hepatitis B virus,
receiving Rituxan could cause the virus to become an active infection
again. Hepatitis B reactivation may cause serious liver problems,
including liver failure and death. You should not receive Rituxan if
you have active hepatitis B liver disease. Your healthcare provider
will monitor you for hepatitis B infection during and for several
months after you stop receiving Rituxan
Progressive Multifocal Leukoencephalopathy
(PML): PML is a rare, serious brain infection
caused by a virus that can happen in people who receive Rituxan.
People with weakened immune systems can get PML. PML can result in
death or severe disability. There is no known treatment, prevention,
or cure for PML

Before receiving Rituxan, patients should tell their healthcare
provider if they:

have had a severe reaction to Rituxan or a rituximab product
currently have or have a history of other medical conditions,
especially heart disease
have had a severe infection, currently have an infection, or have a
weakened immune system
have had a recent vaccination or are scheduled to receive vaccinations
are pregnant or planning to become pregnant. Females who are able to
become pregnant should use effective birth control (contraception)
during treatment with Rituxan and for 12 months after the last dose of
Rituxan
are breastfeeding or plan to breastfeed. Patients should not
breastfeed during treatment and for at least 6 months after the last
dose of Rituxan
are taking any medications, including prescription and
over-the-counter medicines, vitamins, and herbal supplements

What are the possible side effects of Rituxan?

Rituxan can cause serious and life‐threatening side effects, including:

Tumor Lysis Syndrome (TLS): TLS is caused by the fast
breakdown of cancer cells. TLS can cause you to have kidney failure
and the need for dialysis treatment or may cause an abnormal heart
rhythm. Tell your healthcare provider right away if you have any of
the following signs or symptoms of TLS: nausea, vomiting, diarrhea, or
lack of energy
Serious Infections: Serious infections can happen during
and after treatment with Rituxan and can lead to death. Rituxan can
increase your risk of getting infections and can lower the ability of
your immune system to fight infections. People with serious infections
should not receive Rituxan
Heart Problems: Rituxan may cause chest pain, irregular
heartbeats, and heart attack. Your healthcare provider may monitor
your heart during and after treatment with Rituxan if you have
symptoms of heart problems or have a history of heart problems
Kidney Problems: especially if you are receiving Rituxan
for non–Hodgkin’s lymphoma (NHL). Your healthcare provider should do
blood tests to check how well your kidneys are working
Stomach and Serious Bowel Problems That Can Sometimes Lead to
Death: Tell your healthcare provider right away if you have
any stomach-area pain during treatment with Rituxan

What are the most common side effects during treatment with Rituxan?

The most common side effects of Rituxan include:

infusion-related reactions
infections (may include fever, chills)
body aches
tiredness
nausea

In patients with GPA or MPA, the most common side effects of Rituxan
also include:

low white and red blood cells
swelling
diarrhea
muscle spasms

Other side effects include:

aching joints during or within hours of receiving an infusion
more frequent upper respiratory tract infections

These are not all of the possible side effects with Rituxan. For more
information, ask a doctor or pharmacist.

Contact a doctor for medical advice about side effects. Report side
effects to the FDA at (800) FDA‐1088 or http://www.fda.gov/medwatch.
Patients may also report side effects to Genentech at (888) 835‐2555.

Please see the Rituxan Prescribing Information and Medication Guide
including Most Serious Side Effects for additional Important Side Effect
Information at http://www.rituxan.com.

Genentech and Biogen collaborate on Rituxan in the United States, and
Roche markets MabThera in the rest of the world, except Japan, where
Rituxan is co-marketed by Chugai and Zenyaku Kogyo Co. Ltd.

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.

References

1.Medscape. Granulomatosis with Polyangiitis (Wegener
Granulomatosis). Available at: https://emedicine.medscape.com/article/332622-overview#showall
[Last accessed: March 25, 2019]

2. Brogan P, et al. Pediatric Open-Label Clinical Study of Rituximab for
the Treatment of Granulomatosis with Polyangiitis (GPA) and Microscopic
Polyangiitis (MPA) [abstract]. Arthritis Rheumatol. 2018; 70 (suppl 10).

3. Genetic and Rare Diseases Information Center (GARD), National Center
for Advancing Translational Sciences (NCATS), National Institutes of
Health (NIH). ANCA-associated vasculitis. Available at: https://rarediseases.info.nih.gov/diseases/13011/anca-associated-vasculitis
[Last accessed: March 25, 2019]

4. Medscape. Microscopic Polyangiitis. Available at: https://emedicine.medscape.com/article/334024-overview#showall
[Last accessed: March 25, 2019]

5. Iudici M, et al. Childhood-onset granulomatosis with polyangiitis and
microscopic polyangiitis: systematic review and meta-analysis. Orphanet
J Rare Dis. 2016; 11: 141.