Last week, pharmaceutical company Sanofi received a double dose of good news from European regulators about its multiple sclerosis (MS) medications.
Lemtrada (alemtuzumab) won approval and regulators reversed an earlier decision not to grant Aubagio (teriflunomide) “new active status” (NAS) because it is similar to an older drug. Without NAS designation, Aubagio could have faced generic competition in Europe in as little as three years.
This is great news for those who suffer from MS as it adds to the arsenal of drugs to combat this chronic, often disabling, disease.
Multiple sclerosis is thought to be an autoimmune disease that affects the central nervous system. In MS patients, the immune system sees the protective coating of nerve cells—like the plastic coating on a lamp cord—as something foreign to be destroyed.
T-cells eat holes in this coating, called myelin, leaving bare spots where scars or “plaques” form. The bare nerves can no longer effectively transmit impulses and the signal breaks down or shorts out like a defective lamp cord. Depending on where the plaques occur, this loss of conduction can cause a wide array of symptoms ranging from mild numbness and tingling, to complete paralysis or blindness.
Fortunately, there has been an explosion in the MS drug market over the past few years with many more options finding their way into the neurologist’s toolkit. Lemtrada and Aubagio are two of the newest examples.
New Hope via IV Needle
Although more publicity and excitement has been generated over the oral medications coming out, Lemtrada has proven to be a powerful weapon in the fight against MS. Lemtrada has a different method of delivery over other drugs in that it is given intravenously only twice a year. The first dose is administered over a five-day period, with the second dose, six months later, given over a three-day span. So while it still involves the use of a needle, it is neither self-administered nor does it involve a routine that must be adhered to by the patient.
In two phase III trials, Lemtrada was compared to Rebif, a well established MS therapy already on the market. Results showed that Lemtrada demonstrated superior effectiveness. Genzyme (purchased by Sanofi in 2011) made the announcement in a press release on June 28.
“[The] announcement from Genzyme represents a key milestone in the extensive program evaluating LEMTRADA in multiple sclerosis,” said Professor Alastair Compston, head of the Department of Clinical Neurosciences at the University of Cambridge, United Kingdom. “The superior efficacy of Lemtrada vs. Rebif in the clinical trials, which was sustained despite infrequent administration, represents an approach to treatment that promises to reshape the future for many people with active relapsing-remitting multiple sclerosis.”
The results showed that 65 percent of patients treated with Lemtrada were free of relapses at two years, as compared to 47 percent with Rebif. The data also showed a nearly 50 percent reduction in relapse rate as compared to Rebif.
Trials and Tribulations
But it hasn’t been an effortless journey for Lemtrada. Getting the nod earlier this year from the U.S. Food and Drug Administration (FDA) ended nearly a quarter century-long saga for the drug as it made its way to market. Lemtrada started life as Campath-1H when it was originally synthesized in 1983 by Herman Waldmann at Cambridge University’s Department of Pathology (hence the name). The drug was originally intended for use in leukemia patients but began trials in MS in the early 1990s. Campath has been approved as a drug to treat cancer since 2001.
Studies with MS patients showed such promise that neurologists, who only had a handful of disease modifying medications (DMDs) at their disposal at the time, began prescribing it “off-label” for MS patients who were failing other therapies in an attempt to control their disease.
In an unprecedented move to stop this off-label use of the drug, Sanofi pulled Campath from the market in 2011, effectively stopping neurologists from prescribing it to MS patients who were depending on it to control their MS.
Campath changed hands many times before Genzyme (along with Sanofi who purchased the company in 2011), finally saw the success of getting it to market. Now neurologists can prescribe it under the Lemtrada name, designated as a MS therapy.
Aubagio (teriflunomide), Sanofi’s other DMD for MS patients, is an oral drug that contains the same active ingredient as leflunomide, which has been used to treat rheumatoid arthritis since 1998. This caused EU regulators to initially deny the drug NAS designation, which could have meant generic competition for the drug in the European market by 2016. With the reversal of this decision by EU regulators, however, Sanofi can keep the patent on Aubagio for the next 20 years.
Bitter Pill for Those Without Coverage
With all of the new MS therapies hitting the market recently, starting with Novartis’ Gilenya (fingolimod) which won approval in late 2010, this means that patients who have been failing other treatments are granted new hope that one of these therapies might bring them sweet relief from unrelenting relapses. Unfortunately for the patient, all of the therapies are protected with 20-year drug patents until at least 2030. For those with no health insurance, the proliferation of new therapies comes as a bitter pill to swallow.
Patient assistance programs exist to help those whose insurance plans don’t cover the new DMDs or they have no insurance at all. These programs help with generous co-pays, and even cover the entire cost of the medication in some cases. Learn more about assistance programs by visiting the Multiple Sclerosis Association of America.