Market Overview

FDA Grants Priority Review for Genentech's Rituxan (Rituximab) for Pemphigus Vulgaris

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  • Pemphigus vulgaris (PV) is a life-threatening, autoimmune condition
    with limited treatment options
    1
  • The FDA previously granted Breakthrough Therapy Designation and
    Orphan Drug Designation for Rituxan in PV

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) for the treatment of pemphigus vulgaris (PV), a rare,
life-threatening condition characterized by progressive painful
blistering of the skin and mucous membranes.1

The FDA previously granted Breakthrough Therapy Designation and Orphan
Drug Designation to Rituxan for the treatment of PV. Presently, there
are limited approved treatment options available for patients with PV.1

"We are committed to developing medicines for rare diseases with limited
treatment options, such as pemphigus vulgaris," said Sandra Horning,
M.D., chief medical officer and head of Global Product Development. "We
look forward to continued work with the FDA to hopefully provide
patients with a new treatment for this serious and potentially
life-threatening disease."

The sBLA submission is based on data from a Roche-supported randomized
trial conducted in France which evaluated Rituxan plus a tapering
regimen of low dose oral corticosteroid (CS) treatment compared to a
standard dose of CS alone as a first-line treatment in patients with
newly diagnosed moderate to severe pemphigus.2 Results of the
study show that Rituxan provides substantial improvement in pemphigus
vulgaris remission rates and successful tapering and/or cessation of CS
therapy.2 These results were published in The Lancet
in March 2017. Genentech is currently conducting another Phase III study
in PV which is evaluating Rituxan plus a tapering regimen of CS compared
to Cellcept (PEMPHIX, NCT02383589).3

About Pemphigus Vulgaris

Pemphigus vulgaris is an autoimmune, intraepidermal, blistering disease
affecting the skin and mucous membranes.1 This rare,
life-threatening condition is the most common type of a group of
autoimmune disorders collectively called pemphigus, which affect 30,000
to 40,000 people in the United States.4

What 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 GPA and
MPA.

People with serious infections should not receive Rituxan. 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 Reactions: Infusion reactions
    are the most common side effect of Rituxan treatment. Serious infusion
    reactions can happen during an infusion or within 24 hours after an
    infusion
  • Severe Skin and Mouth Reactions: painful
    sores or ulcers on skin, lips, or in the mouth; blisters, peeling
    skin, rash, or pustules
  • Hepatitis B Virus (HBV) Reactivation: If
    a patient has had hepatitis B or is a carrier of hepatitis B virus,
    receiving Rituxan could cause the virus to become an active infection
    again
  • Progressive Multifocal Leukoencephalopathy (PML):
    a rare, serious brain infection caused by the JC virus

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 kidney failure and the need for dialysis
    treatment or may cause an abnormal heart rhythm
  • Serious Infections: Serious infections can happen during and after
    treatment with Rituxan and can lead to death
  • Heart Problems: Rituxan may cause chest pain and irregular heartbeats,
    which may need treatment, or a patient's doctor may decide to stop
    treatment with Rituxan
  • Kidney Problems: especially if a patient is receiving Rituxan for
    non-Hodgkin's lymphoma (NHL). Doctors should do blood tests to check
    how well a patient's kidneys are working
  • Stomach and Serious Bowel Problems That Can Sometimes Lead to Death:
    Patients should inform their doctor right away if they have any
    stomach area pain during treatment with Rituxan
  • Low Blood Cell Counts: A doctor may do blood tests during treatment
    with Rituxan to check a patient's blood cell counts

What are common side effects during treatment with Rituxan?

  • Infusion reactions
  • Chills
  • Infections
  • Body aches
  • Tiredness
  • Low white blood cell counts

Other side effects include:

  • Aching joints during or within hours of receiving an infusion
  • More frequent upper respiratory tract infections

Patients should tell their doctor about any side effect that bothers
them or does not go away.

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. Pemphigus Vulgaris. Available at: http://emedicine.medscape.com/article/1064187-overview.
[Last accessed: January 24, 2018]

2 Joly P, et al. First-Line Rituximab Combined with
Short-Term Prednisone Versus Prednisone Alone for the Treatment of
Pemphigus (Ritux 3): A Prospective, Multicentre, Parallel-Group,
Open-Label Randomised Trial. The Lancet. March 22, 2017

3 ClinicalTrials.gov. A Randomized, Double-Blind Study to
Evaluate the Efficacy and Safety of Rituximab Versus MMF in Patients
With Pemphigus Vulgaris. Available at: https://clinicaltrials.gov/ct2/show/NCT02383589?term=pemphigus+vulgaris&recr=Open&rank=2.
[Last accessed: January 24, 2018]

4International Pemphigus & Pemphigoid Foundation. Pemphigus.
Available at: http://www.pemphigus.org/research/clinically-speaking/pemphigus/.
[Last accessed: January 24, 2018]

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