VICTORIA, B.C., CANADA AND NUTLEY, NJ, USA, Jan. 3 /PRNewswire-FirstCall/ - Aspreva Pharmaceuticals Corporation and Roche today announced that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation for CellCept(R) (mycophenolate mofetil) in the treatment of myasthenia gravis (MG). Aspreva is currently evaluating CellCept for the treatment of myasthenia gravis in a global phase III study.
“We are pleased to have received this orphan drug designation for CellCept in the treatment of myasthenia gravis,” said Richard Glickman, Aspreva’s Chairman and Chief Executive Officer. “This designation is an important step in our clinical development of CellCept for MG and in our efforts to deliver an effective, evidence-based treatment option for this debilitating and life-threatening illness.”
The randomized, double-blind, placebo-controlled clinical trial now in progress is designed to evaluate the efficacy and safety of CellCept in maintaining or improving symptom control with reduced doses of corticosteroids in patients with myasthenia gravis over a treatment period of 36 weeks. The primary endpoints in the trial include both minimal disease activity and low steroid dose. Patient enrolment in the study is complete, and Aspreva expects to conclude the trial in late 2006.
“Roche and Aspreva are highly committed to our partnership, to CellCept, and to developing better treatments for patients with a range of autoimmune diseases,” said Dennis Burns, Roche’s Global Head of Business Development. “With the potential to use CellCept in myasthenia gravis, we may be able to offer patients with few options a chance for better outcomes.”
The FDA’s orphan drug designation is intended to encourage the development of new treatments for rare diseases. It is granted for treatments designed to prevent, diagnose or treat rare, life-threatening or chronically debilitating diseases that affect fewer than 200,000 people in the United States.
About Myasthenia Gravis
According to the Myasthenia Gravis Foundation, myasthenia gravis (MG) affects approximately 70,000 to 100,000 people worldwide, including approximately 36,000 people in the United States. MG is a debilitating, chronic autoimmune neuromuscular disease in which the body produces auto- antibodies which prevent the nerves from sending messages to the muscles.
Although MG can affect any voluntary muscle, it frequently involves those controlling eye movements, chewing, swallowing, coughing and facial expressions, but can be more severe in some patients.
Complete remission is infrequent and long-term immunosuppression is usually required. Current treatments for MG include the use of cholinesterase inhibitors, steroids and other immunosuppressant drugs such as azathioprine.
About CellCept
CellCept is Roche’s leading immunosuppressant or “anti-rejection” drug, used in combination with other immunosuppressive drugs (cyclosporine and corticosteroids) for the prevention of rejection in patients receiving heart, kidney and liver transplants.
CellCept was first approved for use in combination therapy for the prevention of acute organ rejection in kidney transplantation in 1995 and has since been approved worldwide for prevention of organ rejection in adult kidney, heart and liver transplantation. In some countries, it has also been approved for pediatric kidney transplantation. This therapeutic success represents 10 years of clinical experience and patient benefits, including reduced toxicities and prolonged organ and patient survival. Over the last decade, CellCept has become one of the most widely studied immunosuppressants and research is ongoing both in organ transplantation and related areas, such as autoimmune disease, to help provide clinical benefit to a wider range of patients.
In October 2003, Aspreva announced a collaboration agreement with Roche for the exclusive worldwide rights (excluding Japan) to develop and, upon regulatory approval, commercialize CellCept for all autoimmune disease applications.
CellCept is not currently approved for the treatment of any autoimmune disease including myasthenia gravis.
There are no adequate and well-controlled studies of CellCept in pregnant women. As CellCept has been shown to have teratogenic effects in animals at subclinical doses on a body surface area basis, it may cause fetal harm when administered to a pregnant woman. CellCept should not be used in pregnant women unless the potential benefit justifies the potential risk to the fetus. Women of childbearing potential should have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL within one week prior to beginning therapy even where there has been a history of infertility, unless due to hysterectomy.
Women of childbearing potential must use effective contraception before beginning CellCept therapy, during therapy and for six weeks following discontinuation of therapy. Two reliable forms of contraception must be used simultaneously unless abstinence is the chosen method. If pregnancy occurs during treatment, the physician and patient should discuss the desirability of continuing the pregnancy (see complete product information).
Adverse events reported in greater than 30% of renal, cardiac or liver transplant patients receiving CellCept (in combination with cyclosporine and corticosteroids) were pain, fever, headache, asthenia, anemia, leucopenia (patients should be monitored for neutropenia; dosing should be interrupted or the dose reduced if neutropenia develops), thrombocytopenia, leukocytosis, urinary tract infection, hypertension, hypotension, peripheral edema, hypercholesteremia, hypokalemia, hyperglycemia, creatinine, BUN and cough increased, hypomagnesemia, diarrhea, constipation, nausea, vomiting, respiratory infection, dyspnea, lung disorder, pleural effusion, tremor and insomnia.
Patients receiving immunosuppressant regimens are at increased risk of developing lymphomas and other malignancies, particularly of the skin.
Increased susceptibility to infection and the possible development of lymphoma may result from immunosuppression. Only physicians experienced in immunosuppressive therapy and management of renal, cardiac or hepatic transplant patients should use CellCept. Patients receiving the drug should be managed in facilities equipped and staffed with adequate laboratory and supportive medical resources. The physician responsible for maintenance therapy should have complete information requisite for the follow-up of the patient. For full prescribing information on CellCept, please visit: www.rocheusa.com/products/cellcept.
About Aspreva Pharmaceuticals
Aspreva is an emerging pharmaceutical company focused on identifying, developing and, upon regulatory approval, commercializing new indications for approved drugs and late stage drug candidates for patients living with less common diseases. Aspreva’s “Indication Partnering” strategy allows its partners to maintain core brand focus while extending the benefits of their medicines to a broader patient population. Aspreva is listed on the NASDAQ National Market under the trading symbol “ASPV” and on the Toronto Stock Exchange under the trading symbol “ASV”. For further information on Aspreva, please visit www.aspreva.com.
About Roche
Founded in 1896 and headquartered in Basel, Switzerland, Roche is one of the world’s leading innovation-driven healthcare groups. Its core businesses are pharmaceuticals and diagnostics. Roche is one of the world’s leaders in diagnostics, the leading supplier of pharmaceuticals for cancer, as well as a leader in virology and transplantation. As a supplier of products and services for the prevention, diagnosis and treatment of disease, the Group contributes on many fronts to improve people’s health and quality of life. Roche employs roughly 65,000 people in 150 countries, including approximately 15,000 in the United States. For further information, please visit Roche’s worldwide and U.S. websites (Global: www.roche.com and U.S.: www.rocheusa.com).
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Act of 1995. These include without limitation statements related to our clinical trials, product candidates and operations. Words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward- looking statements contain these identifying words. These forward-looking statements are based upon our current expectations and we may not actually achieve the plans, approvals, intentions or expectations disclosed in our forward-looking statements. Forward-looking statements involve risks and uncertainties. Our actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks related to the progress, timing and results of our clinical trials, our ability to attract and retain collaborations relating to the development and commercialization of new indications, intellectual property matters, difficulties or delays in obtaining regulatory approval, the FDA may finally determine that the design and planned analysis of our clinical trail do not adequately address the trial objectives in support of our regulatory submission, competition from other pharmaceutical or biotechnology companies, and other risks detailed in our filings with the Securities and Exchange Commission and Canadian securities regulatory authorities. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. All forward-looking statements are qualified in their entirety by this cautionary statement, and Aspreva undertakes no obligation to revise or update any forward-looking statements as a result of new information, future events or otherwise after the date hereof.
CONTACTS: Sage Baker Director, Corporate Communications Aspreva Pharmaceuticals (250) 744-2488 ext. 270 sbaker@aspreva.com Maureen Byrne Director, Public Affairs Roche (973) 562-2203 maureen.byrne@roche.com
Aspreva Pharmaceuticals
CONTACT: Sage Baker, Director, Corporate Communications, AsprevaPharmaceuticals, (250) 744-2488 ext. 270, sbaker@aspreva.com; MaureenByrne, Director, Public Affairs, Roche, (973) 562-2203,maureen.byrne@roche.com
