FDA Grants Priority Review for Genentech’s OCREVUS™ (ocrelizumab) Biologics License Application
• Genentech announced the FDA has accepted the filing application for OCREVUS (ocrelizumab) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS) and granted the application Priority Review Designation with a targeted action date of December 28 2016. The EMA has also validated the filing application.
• If approved by the FDA and EMA for both indications, OCREVUS would be the first and only treatment indicated for both forms of MS, which affect approximately 95 percent of people at diagnosis. Currently, there are no approved medicines for PPMS, a debilitating form of MS.
• Regulatory applications for ocrelizumab are based on positive results from three Phase III studies, which met primary and key secondary endpoints.
June 28, 2016 01:00 AM Eastern Daylight Time
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) accepted for review the company’s Biologics License Application (BLA) for OCREVUS™ (ocrelizumab) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS), and granted the application Priority Review Designation with a targeted action date of December 28, 2016.
The OCREVUS Marketing Authorization Application (MAA) has also been validated by the European Medicines Agency (EMA). If approved by the FDA and EMA for both indications, OCREVUS would be the first and only treatment indicated for both forms of MS, which affect approximately 95 percent of people at diagnosis.
“OCREVUS is the first investigational medicine to significantly reduce disability progression in people with relapsing and primary progressive forms of MS,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We are pleased by the FDA’s decision to classify their review of the BLA as priority because we believe OCREVUS has the potential to help people living with either of these two forms of MS. We will continue to work closely with the FDA and EMA to bring this investigational medicine to people with MS as quickly as possible.”
Priority Review Designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the safety and effectiveness of the treatment of a serious disease. In February 2016, the FDA granted Breakthrough Therapy Designation to OCREVUS for the treatment of PPMS. OCREVUS is the first investigational medicine to receive Breakthrough Therapy Designation in MS.
The OCREVUS marketing applications are based on positive results from three Phase III studies, which met primary and key secondary endpoints. Data from two identical studies (OPERA I and OPERA II) in people with RMS showed superior efficacy of OCREVUS in reducing annualized relapse rates and disability progression sustained for at least three and for at least six months compared with Rebif® (interferon beta-1a). Data from the ORATORIO study in people with PPMS showed significant reductions in disability progression sustained for at least three and for at least six months, as well as in as other measures of progressive disease compared with placebo. Overall safety (proportion of patients with adverse events and serious adverse events) of OCREVUS in the Phase III studies was similar to interferon beta-1a in the RMS studies and to placebo in the PPMS study. The most common adverse events associated with OCREVUS were infusion-related reactions and infections, which were mostly mild to moderate in severity.
OCREVUS™ is the proprietary name submitted to U.S. Food and Drug Administration (FDA) for the investigational medicine ocrelizumab.
About OCREVUS™ (ocrelizumab)
OCREVUS is an investigational, humanized monoclonal antibody designed to selectively target CD20-positive B cells, a specific type of immune cell thought to be a key contributor to myelin (nerve cell insulation and support) and axonal (nerve cell) damage. This nerve cell damage can lead to disability in people with MS. Based on preclinical studies, OCREVUS binds to CD20 cell surface proteins expressed on certain B cells, but not on stem cells or plasma cells, and therefore important functions of the immune system may be preserved.
The Phase III clinical development program for OCREVUS (ORCHESTRA) includes three studies: OPERA I, OPERA II and ORATORIO. OPERA I and OPERA II are identical Phase III, randomized, double-blind, double-dummy, global multi-center studies that evaluated the efficacy and safety of OCREVUS (600 mg administered by intravenous infusion every six months) compared with interferon beta-1a (44 mcg administered by subcutaneous injection three times per week) in 1,656 people with relapsing forms of MS (i.e., relapsing-remitting MS and secondary-progressive MS with relapses).1 ORATORIO is a Phase III, randomized, double-blind, global multi-center study that evaluated the efficacy and safety of OCREVUS (600 mg administered by intravenous infusion every six months) compared with placebo in 732 people with primary progressive MS (PPMS).2
About multiple sclerosis
Multiple sclerosis (MS) is a chronic disease that affects an estimated 2.3 million people around the world, for which there is currently no cure.3,4 MS occurs when the immune system abnormally attacks the insulation and support around nerve cells (myelin sheath) in the brain, spinal cord and optic nerves, causing inflammation and consequent damage. This damage can cause a wide range of symptoms, including muscle weakness, fatigue and difficulty seeing, and may eventually lead to disability.5,6,7 Most people with MS experience their first symptom between 20 and 40 years of age, making the disease the leading cause of non-traumatic disability in younger adults.8
Relapsing MS is the most common form of the disease. Disease activity and progression can occur even when people do not show signs or symptoms of MS, despite available relapsing MS treatments. Primary progressive MS (PPMS) is a debilitating form of the disease marked by steadily worsening symptoms but typically without distinct relapses or periods of remission.9 Approximately one in 10 people with MS are diagnosed with the primary progressive form of the disease. There are no approved treatments for PPMS.
About Genentech in neuroscience
Neuroscience is a major focus of research and development at Genentech and Roche. The company’s goal is to develop treatment options based on the biology of the nervous system to help improve the lives of people with chronic and potentially devastating diseases. Roche has more than a dozen investigational medicines in clinical development for diseases that include multiple sclerosis, Alzheimer’s disease, spinal muscular atrophy, Parkinson’s disease, Down syndrome and autism.
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.
All trademarks used or mentioned in this release are protected by law. Rebif is a registered trademark of Merck KGaA and EMD Serono, Inc.
References
1 F. Hoffmann-La Roche. ClinicalTrials.gov NCT01247324 and NCT01412333. National Library of Medicine. Available at: https://clinicaltrials.gov/ct2/show/NCT01247324 and https://clinicaltrials.gov/ct2/show/NCT01412333.
2 F. Hoffmann-La Roche. ClinicalTrials.gov NCT01194570. National Library of Medicine. Available at: https://clinicaltrials.gov/ct2/show/NCT01194570.
3 Multiple Sclerosis International Federation. (2013). Atlas of MS 2013. Available at: http://www.msif.org/about-us/advocacy/atlas/.
4 National Institutes of Health-National Institute of Neurological Disorders and Stroke. (2015). Multiple Sclerosis: Hope Through Research. Available at: http://www.ninds.nih.gov/disorders/multiple_sclerosis/detail_multiple_sclerosis.htm#280373215.
5 Ziemssen T. (2005). Modulating processes within the central nervous system is central to therapeutic control of multiple sclerosis. J Neurol, 252(Suppl 5), v38-v45.
6 Hauser S.L. et al. (2012). Multiple sclerosis and other demyelinating diseases. In Harrison’s Principles of Internal Medicine (pp.3395-3409). New York, NY: McGraw Hill Medical.
7 Hadjimichael O. et al. (2007). Persistent pain and uncomfortable sensations in persons with multiple sclerosis. Pain, 127(1-2), 35-41.
8 Multiple Sclerosis International Federation. What is MS? Available at http://www.msif.org/about-ms/what-is-ms/. Last accessed January 2015.
9 MS International Federation. Types of MS. Available at: http://www.msif.org/about-ms/types-of-ms/.
Contacts
Genentech
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Kimberly Muscara, 650-467-6800
Investor Contacts:
Neera Ravindran, 650-491-5281
Karl Mahler, 011 41 61 687 8503
