NEWS 07/23/1993
P93-31 Food and Drug Administration FOR IMMEDIATE RELEASE Monica Revelle (301) 443-4177 The Food and Drug Administration today announced the licensing of Interferon beta-1b, the first product for the treatment of certain patients with multiple sclerosis and the first biotechnology product to be licensed under FDA's accelerated approval regulations. Multiple sclerosis is a chronic, often disabling disease of the central nervous system that occurs when a protective sheath surrounding the nerve fibers breaks down. Nearly 30 percent of multiple sclerosis patients suffer from a relapsing-remitting form of the disease in which symptoms disappear totally or partially after a flare-up and are followed by a period of stability that can last for months or years. A clinical trial involving 372 patients with relapsing- remitting multiple sclerosis indicated that administration of Interferon beta-1b by injection every other day decreased the frequency of flare-ups and kept more patients free of flare-ups over a two-year treatment period. "This product marks an important first," said FDA Commissioner David A. Kessler, M.D. "It's not a cure, but it will help relieve debilitating symptoms for many multiple sclerosis patients for whom no other relief is available. "We gave this drug a top-priority review," he said. "This demonstrates our commitment to act quickly to make drugs available to those who need them." The accelerated approval policy allows for expediting the approval of therapies that provide a meaningful therapeutic benefit for patients with serious illnesses. It enables FDA to approve therapies as soon as their safety and effectiveness can be reasonably established. Accelerated approval relies solely or in part on "surrogate endpoints" -- laboratory measurements or physical signs -- for evidence of effectiveness. The surrogate endpoints are believed to be likely to predict benefit for the patient. The surrogate endpoints used in licensing Interferon beta-1b were data from magnetic resonance imaging (MRI) scans of the brain, which supported the clinical findings. MRI scans indicated that after two years of treatment, there was a greater increase in the multiple sclerosis lesion areas in the brains of patients treated with a placebo than in patients treated with Interferon beta-1b. The trials did not demonstrate, however, that the findings on MRI scans correlate with slower disease progression. The accelerated approval regulations require the product's manufacturer, Chiron Corp. of Emeryville, Calif., to conduct postmarketing studies to investigate the effectiveness of Interferon beta-1b in slowing or preventing progression of multiple sclerosis. Adverse reactions following administration of Interferon beta- 1b included inflammation and pain at the injection site and flu- like symptoms. The use of other types of interferon has also been associated with occasional serious side effects including abnormal liver function tests and severe depression, including suicidal depression. These events were also observed in multiple sclerosis patients taking Interferon beta-1b in clinical trials. An estimated 250,000 to 350,000 Americans are affected by multiple sclerosis, which affects twice as many women as men. Two thirds of the patients experience their first symptoms between the ages of 20 and 40. On March 19, the Peripheral and Central Nervous System Drugs Advisory Committee recommended approval of Interferon beta-1b for decreasing the frequency of flare-ups in patients with relapsing- remitting multiple sclerosis. On June 3, FDA sent an "approvable" letter that informed Chiron Corp. that the agency was prepared to approve the product when certain conditions were met. Chiron then submitted the requested information and made a commitment to conduct postmarketing studies. Interferon beta-1b is manufactured by Chiron under the trade name "Betaseron." Berlex Laboratories of Alameda, Calif., will distribute it. FDA is one of eight Public Health Service agencies within HHS. MILESTONES IN BETASERON APPROVAL PROCESS Betaseron was licensed under the accelerated approval policy. o Biologics licensed in the normal process take an average of 28 months for FDA review of the Product License Application (PLA). The FDA review of Betaseron, including intensive discussions and presentation of more data, took just 12 months. o Betaseron is the first biotechnology product licensed under the accelerated approval process. It was preceded by DDC, a drug for AIDS, which was approved under the same program last year. o The review of the PLA for Betaseron was the first collaborative effort on a marketing application by the Center for Biologics Evaluation and Research and the Center for Drug Evaluation and Research. IMPORTANT DATES March 3, 1983 Investigational New Drug Application filed Nov. 11, 1988 FDA designates Betaseron as an Orphan Drug July 23, 1992 Chiron Corp. files the PLA with FDA March 19, 1993 Peripheral and Central Nervous System Drugs Advisory Committee recommends approval May 30, 1993 FDA completes its review of the PLA June 3, 1993 FDA sends Chiron "Approvable Letter" July 23, 1993 FDA licenses Betaseron