BASEL, Switzerland—Novartis AG won approval from the European Commission for Gilenya (fingolimod) 0.5 mg daily as a disease-modifying therapy in patients with highly active relapsing-remitting multiple sclerosis (RRMS) despite treatment with beta interferon, or in patients with rapidly evolving severe RRMS—making it the first approved oral MS treatment in the European Union (EU). The approval was based on the largest clinical trial program submitted to date for a new multiple sclerosis drug, and included 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.

David Epstein, division head of Novartis Pharmaceuticals, says that the announcement marks another "major regulatory approval and we are pleased that Gilenya will become available to more eligible MS patients. Novartis is dedicated to bringing innovative new treatments to patients where there is significant unmet need. Gilenya has been in clinical development for MS since 2003 and we are grateful for the commitment of those involved, especially the trial participants, who have contributed significantly to the development of this novel medicine."

This approval follows the news in September 2010 that the U.S. Food and Drug Administration (FDA) had approved the drug for relapsing forms of MS—the most common forms of the disease.

Gilenya, the first in a new class of drugs called sphingosine 1-phosphate receptor (S1PR) modulators, was licensed from Mitsubishi Tanabe Pharma Corp. The novel mechanism of Gilenya is thought to work by reducing the immune system's attack on the central nervous system in MS patients by retaining certain white blood cells (lymphocytes) in the lymph nodes. This prevents the white blood cells from reaching the central nervous system, where they could potentially attack the protective covering around the nerve fibers, resulting in less inflammatory damage to the nerve cells. The white blood cell retention is reversible if Gilenya treatment is stopped.

The EU application included data showing that this approved dosage of Gilenya reduced relapses by 52 percent at one year compared with interferon beta-1a IM (Avonex), one of the most commonly prescribed treatments for MS. Data from a two-year placebo-controlled study showed a 30 percent reduction in the risk of disability progression among Gilenya patients compared with placebo), and clinical studies also indicated that treatment with Gilenya resulted in "statistically significant" reductions in brain lesion activity as measured by MRI.

Also statistically significant could be the payoff from prescriptions of the drug, with healthcare analyst Trung Huynh at Datamonitor noting: "European approval is set to boost sales another $900 million as Novartis become the leading player in this lucrative multibillion dollar market."

Some estimates by analysts point to the MS market growing to $15 billion by 2015, and many expect that Novartis will be one of the major beneficiaries of this growth because, as some have noted, many newly diagnosed MS patients will likely begin therapy with Gilenya rather than with older treatments.

Labels: Gilenya