The European Medicines Agency (EMA) has accepted the filing of Genzyme's marketing authorization application (MAA) for once-daily oral teriflunomide for the treatment of relapsing forms of multiple sclerosis (MS). Acceptance of the MAA starts the EMA’s review process.
Teriflunomide is an immunomodulatory, disease-modifying oral drug with anti-inflammatory properties, and is under investigation for the treatment of MS. Teriflunomide blocks the proliferation and functioning of activated T and B lymphocytes – which are thought to be especially damaging in MS – by selectively and reversibly inhibiting a mitochondrial enzyme. Slowly dividing or resting lymphocytes are generally unaffected by teriflunomide, suggesting that the immune system’s response to infection should not be compromised.
“The filing represents another important milestone for teriflunomide and brings us one step closer to offering a new treatment option to patients with relapsing MS,” said Bill Sibold, senior vice president, head of multiple sclerosis, Genzyme. “As an oral therapy with a promising clinical profile, teriflunomide is extremely well positioned to provide an alternative therapeutic option to patients who are currently taking injectable therapies. Those injectable therapies make up approximately 80 per cent of the MS market today.”
The purpose of the MAA is to secure approval to market and allow prescription of teriflunomide in the European Union based on data from two completed pivotal phase III trials, TEMSO and TENERE. These trials represent two of five efficacy studies of teriflunomide in MS that are completed or underway, making the clinical program one of the largest and broadest of any MS therapy in development.
An application to market teriflunomide in the United States is under review by the US Food & Drug Administration.
Teriflunomide is being studied in a large clinical program that is expected to include more than 5,000 trial participants in 36 countries. Five efficacy clinical trials are either completed or underway with teriflunomide, making the clinical program one of the largest and broadest of any MS agent under development. In addition to the TEMSO and TENERE trials, the phase III, placebo-controlled trial TOWER is ongoing in people with relapsing forms of MS. Another Phase III study, TOPIC, is underway in early MS or CIS (clinically isolated syndrome). Teriflunomide is also being evaluated as an adjunct therapy to interferon-ß in the phase III TERACLES trial. With up to 10 years of continuous use in a phase II extension, teriflunomide has the longest clinical experience of any investigational oral MS therapy.
Genzyme, a Sanofi company, has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years.