Thursday, Mar 23, 2017

FDA Grants Breakthrough Therapy Designation for Rituxan® (Rituximab) in Pemphigus Vulgaris

• Pemphigus vulgaris (PV) is a life-threatening, autoimmune condition with limited treatment options ¹
• Genentech is currently enrolling a Phase III clinical trial in patients with moderate to severe PV
• The FDA previously granted Orphan Drug Designation to Rituxan for PV
• Fifteenth Breakthrough Therapy Designation granted to Genentech medicines since 2013

South San Francisco, CA -- March 23, 2017 --

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to Rituxan^® (rituximab) for pemphigus vulgaris, a rare, serious and life-threatening condition characterized by progressive painful blistering of the skin and mucous membranes.² FDA Breakthrough Therapy Designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases and to help ensure that patients receive access to medicines as soon as possible. Genentech is currently enrolling a Phase III study in pemphigus vulgaris (PEMPHIX, NCT02383589), a disease for which there are limited treatment options. ¹

“People with pemphigus vulgaris need more options and we look forward to working with the FDA to make Rituxan available to patients with this potentially deadly disease,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We are committed to developing therapies that target B cells in areas of unmet need across a range of immune and malignant diseases, including new antibody constructs that enhance efficacy, safety or both.”

This is the fifteenth Breakthrough Therapy Designation granted to Genentech medicines since 2013.

Breakthrough Therapy Designation status was granted based on data from a Roche-supported randomized trial conducted in France, which evaluated Rituxan plus oral corticosteroid (CS) treatment compared to CS as a first-line treatment in patients with moderate to severe pemphigus. ³ Results of the study, published in The Lancet, show that Rituxan may provide substantial improvement in pemphigus vulgaris remission rates and successful tapering and/or cessation of CS therapy. ³

In 2015, the FDA granted Orphan Drug Designation to Rituxan for the treatment of pemphigus vulgaris.

About Pemphigus Vulgaris

Pemphigus vulgaris is an autoimmune, intraepidermal, blistering disease affecting the skin and mucous membranes.¹ It is the most common type of a group of autoimmune disorders collectively called pemphigus. ² It is estimated that around three in every 100,000 people are diagnosed with this disease.⁴

About the PEMPHIX study

Genentech initiated a Phase III, randomized, double-blind, double-dummy, active-comparator, parallel-arm multicenter study (PEMPHIX, NCT02383589) to evaluate the efficacy and safety of Rituxan compared with mycophenolate mofetil (MMF) in patients with moderate to severe active pemphigus vulgaris requiring 60-120 mg/day oral prednisone (or equivalent).⁵

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 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or 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: March 7, 2016]

² Healthline. Pemphigus Vulgaris. Available at: http://www.healthline.com/health/pemphigus-vulgaris#Overview1 . [Last accessed: March 7, 2016]

³ Joly P, et al. First Line Rituximab Combined with Short-Term Prednisone Alone for the Treament pf Phemphigus (Ritux3): A Prospective, Multicentre, Parallel-Group, Open-Label Randomised Trial. The Lancet. March 22, 2017

⁴ National Health Service. Pemphigus Vulgaris. Available at: http://www.nhs.uk/Conditions/Pemphigus-vulgaris/Pages/Definition.aspx . [Last accessed: November 24, 2015]

⁵ 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: March 7, 2016]

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