When it comes to drug development, the unmet need of crafting viable, effective Multiple Sclerosis treatments continues to be a major issue. The disease continues to increase throughout the world, with more than two million people presenting with some firm of MS. Most recently, two prominent treatments for Multiple Sclerosis received a vote of no-confidence from governmental drug administrations, indicating that the search for viable MS treatments still has a long way to go.
A report from last week indicated that the U.S. Food and Drug Administration rejected the Multiple Sclerosis treatment Lemtrada, on the basis that the drug has failed to demonstrate a high enough level of efficacy, particularly compared with its potential side effects and danger to the patient. Alemtuzumab, the generic name for the drug, is currently approved in the United States for the treatment for B-cell chronic lymphocytic leukemia (CLL). Used under the trade name under the Campath, even in this application, its use is highly limited for only “compassionate-care” situations. The drug is manufactured by Sanofi via its Genzyme production unit, which was pushing for the FDA to approve the drug for treatment for relapsing forms of MS. “Relapsing-Remitting Multiple Sclerosis” is often associated with the early onset of the disease. The FDA argued that, particularly for this type of Multiple Sclerosis treatment designation, Lemtrada “has not submitted evidence from adequate and well-controlled studies that demonstrate the benefits of Lemtrada outweigh its serious adverse effects,” and has called for a new round of clinical trials for the drug before it will be considered again for public use in the United States. Lemtrada (alemtuzumab) is currently approved in the EU and other countries as a Multiple Sclerosis treatment.
The rejection of Lemtrada is not only a blow for Sanofi, whom analysts believed would rake in close to $500 million in 2014 revenues had the drug been approved by the FDA, but also for the growing numbers of MS patients who are finding that many of the treatment options in the mainstream are simply ineffective or too dangerous to take in order to mitigate symptoms and slow down progression of the disease.
Sanofi also manufactures an oral therapy for MS called Aubagio (teriflunomide), which was cleared by the FDA in 2012 and in Europe at the end of August in 2013. However, recently, this drug too received a vote of no confidence from the likes of a major governmental body. While the drug has been approved in Germany as a therapy for Relapsing-Remitting MS for adults since August 2013, the German Institute for Quality and Efficiency in Health Care (IQWiG) performed an early benefit test on the new drug to see if it offered improved results and fewer side effects compared to the appropriate comparator therapy specified by the Federal Joint Committee (G-BA). Their findings revealed that, while certain side effects are less prevalent when teriflunomide is used compared with an older treatment, such as beta interferon 1a, other serious side effects are in fact are more prevalent. The institute determined that, overall, the drug has not demonstrated a proven benefit. The IQWiG report stipulated: “Regarding disability progression and relapses such as vision disorders, there were no statistically significant differences between the teriflunomide and the interferon group. No statistically significant difference was observed for the outcome ‘health-related quality of life,’ either.”
While few viable treatment options exist for MS sufferers, there are still a considerable number of experimental Multiple Sclerosis therapies in the drug development pipeline that continue to progress through clinical trials toward FDA approval.