The multiple sclerosis drug fingolimod has become the first oral treatment approved in the United States to reduce the frequency of flare-ups and slow disability from relapsing forms of the disease.
The drug, to be marketed as Gilenya in 0.5 mg capsules, represents the first in a new class of drugs called sphingosine 1-phosphate receptor (S1PR) modulators that are able “to block some blood cells in lymph nodes, reducing their migration to the brain and spinal cord, which may help with reducing the severity of MS,” the Food and Drug Administration said Sept. 22 in a written statement.
“Gilenya is the first oral drug that can slow the progression of disability and reduce the frequency and severity of symptoms in MS, offering patients an alternative to currently available injectable therapies,” Dr. Russell Katz, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in the statement.
The FDA approval is good news especially for patients who are weary of treatments involving injections or those who have failed other treatments, Dr. Jonathan Carter, associate professor of neurology at the Mayo Clinic in Scottsdale, Ariz., said in an interview: “We now have an oral therapy option, which has been an unmet need for many years in terms of MS therapies. There are patients who are really intolerant of injections because of needlephobia, or they just can't do it. This offers a treatment option for that group of patients.”
The drug was approved with a Risk Evaluation and Mitigation Strategy (REMS) to educate patients and healthcare providers about safe use and serious risks of fingolimod. Drug manufacturer Novartis will conduct postmarketing research on “selected safety-related outcomes and a voluntary pregnancy registry,” the company said.
Patients taking fingolimod may experience bradycardia or bradyarrhythmia, serious infections, macular edema, and breathing and liver problems. The FDA said the most frequent adverse reactions in clinical trials include headache, influenza, diarrhea, back pain, elevation of certain liver enzymes, and cough.
According to Novartis, Gilenya’s approval was based on the “largest clinical trial program ever submitted to date to the FDA for a new MS drug and included combined data from clinical studies showing significant efficacy in reducing relapses, the risk of disability progression, and the number of brain lesions detected by magnetic resonance imaging (MRI), a measure of disease activity, in people with relapsing forms of MS.”
The company-sponsored clinical trial included data from more than 2,600 patients with more than a total of 4,500 patient-years of experience, Novartis said in its own written statement Sept. 22. The trial also showed “superior efficacy by reducing relapses by 52% at one year compared with interferon beta-1a IM, a commonly prescribed treatment,” Novartis said. Some patients are in their seventh year of treatment, the company noted.
In the United States, MS affects more than 400,000 people and more than 2 million worldwide according to the National Multiple Sclerosis Society.
Russia was the first country to approve Gilenya; the company announced the drug’s 2011 Russian launch earlier this month.
Novartis first submitted Gilenya to the European Medicines Agency (EMA) and to the FDA for review in December 2009. The EMA regulatory review and other filings worldwide are ongoing.
The drug’s prescribing information will be available on the Drugs@FDA Web site, the agency said.