Treatments under FDA Review and Other News
By Susan Wells Courtney
Lemtrada Application Submitted to FDA
As MSAA previously reported, the United States Food and Drug Administration (FDA) had accepted an application in January for the review of Lemtrada™ (alemtuzumab, formerly known as Campath) for the treatment of “relapsing multiple sclerosis.” The FDA will review the efficacy and safety data from the clinical trials; a decision is expected sometime later this year.
Lemtrada is a humanized monoclonal antibody that targets a protein present on the surface of mature lymphocytes, and results in a rapid depletion/suppression of T and B cells. According to Genzyme, their team of scientists “…is also studying whether alemtuzumab, which has completed phase III studies for relapsing-remitting MS, can effectively treat progressive MS, a much more advanced form of the disease.”
A Phase II study of 334 individuals with early, active relapsing-remitting MS (RRMS) compared Lemtrada to high-dose Rebif
(44 mcg). This trial is notable as Lemtrada-treated patients had the lowest relapse rate ever reported for an MS drug.
In a multi-year extension study of the 334 individuals who participated in the original Phase II study, Lemtrada yielded a 73-percent reduction in risk for sustained accumulation of disability, while 77 percent of Lemtrada-treated patients were relapse-free. A five-year assessment showed that 87 percent were free of sustained disability accumulation, 72 percent were relapse-free, and 65 percent were free of clinical-disease activity. These data indicate that Lemtrada’s treatment effect is durable; it halts clinical-disease activity in a significant proportion of RRMS patients through five years – even though many of those patients did not require subsequent re-treatment with the drug.
For more information on this experimental treatment, please see this section of MSAA’s MS Research Update, found at http://mymsaa.org/publications/msresearch-update-2013/lemtrada/ (please note that portions of the information above were originally published in this update).
New Dosing Regimen for Copaxone Submitted to FDA
Teva Pharmaceuticals Industries, Ltd., the makers of Copaxone® (glatiramer acetate), announced on May 30, 2013 that the FDA had accepted a supplemental new drug application for Copaxone at a higher dose and reduced frequency. The present FDA-approved dose of Copaxone is 20 mg given daily via subcutaneous (under the skin) injection.
The new dosing under review is double the concentration (40 mg) and is given three days per week (also via subcutaneous injection) versus every day. Results of the Phase III Glatiramer Acetate Low-frequency Administration (GALA) trial were similar to those seen with the standard daily dose of Copaxone, with no new safety concerns. For more information, please see this section of MSAA’s MS Research Update, found at http://mymsaa.org/publications/msresearch-update-2013/copaxone.
Biogen Idec Submits a New MS Treatment to FDA
On July 19, 2013, Biogen Idec announced that the FDA had accepted their application for the marketing approval of Plegridy™ in the United States. This potential new disease-modifying therapy (DMT) for the long-term treatment of MS is a pegylated version of interferon beta-1a. Plegridy does not need to be taken as often as the presently approved self-injected DMTs for MS, which range from once daily to once weekly. This new medication has been studied in two groups – with injections given subcutaneously either every two weeks or every four weeks. For more information, please see MSAA’s online news article found at http://mymsaa.org/news/new-treatment-plegridy/.
Approval of Phase I Stem Cell Study in Progressive MS
The stem cell research division of the Tisch MS Research Center of New York has announced the FDA approval of a Phase I stem cell study in individuals with progressive MS. The study will use a patient’s own stem cells, taken from his or her bone marrow, in an open label, Phase I clinical trial. According to the center’s news article, approximately 20 progressive MS patients, recruited from the existing patient population of the International Multiple Sclerosis Management Practice (IMSMP), will be initially enrolled.
The article also notes that the Tisch MS Research Center stem cell trial is the first of its kind in the United States and incorporates several key advantages in terms of the type of stem cells being used and how they are administered (via multiple rounds of treatment into the cerebrospinal fluid). The center states that this FDA approval is the culmination of more than a decade of research into the therapeutic potential of stem cells for MS patients. Their initial (“proof of concept”) studies with an animal model of MS showed that the injected stem cells migrated to areas of demyelination and may have affected the rate of repair.
Continued Heart Monitoring Advised during and after Novantrone®
In June 2013, MSAA posted a message online (at mymsaa.org) to warn individuals of the potential danger of Novantrone® (mitoxantrone) to the heart. The purpose of this message is to inform the MS community that the harm to the heart’s pumping action can appear while someone is being treated with the medicine or many years later. Individuals who have, are, or will be taking Novantrone need to have their heart tested before starting treatment and every year thereafter, even after discontinuing the medication. To read the entire message, please go to http://mymsaa.org/
Vaccine Safety and MS
Published studies to date continue to affirm the safety of several vaccinations for individuals with MS. These inactivated vaccines do not increase the risk of developing MS or exacerbating its symptoms. No evidence of a specific risk for relapse has been associated with any of these vaccinations:
- influenza (via the injected flu vaccine using inactivated viruses*)
- hepatitis B
- varicella (chickenpox)
*Please note that flu vaccines are usually available in two forms: injected and intranasal. The injected type of flu vaccine uses the inactivated (or killed) viruses, and is considered safe for individuals with MS. People cannot develop the flu from the injected vaccines, since these contain non-infectious particles. The intranasal vaccine (FluMist), given by nose with a mist, contains live viruses and is NOT recommended for individuals with MS.
Before receiving any vaccine, individuals with MS should consult their physician to make sure that the specific vaccine and its timing is appropriate. If experiencing a relapse, patients may be advised to wait a period of time (approximately four to six weeks) before receiving a vaccine. For more information, please see MSAA’s online article at http://mymsaa.org/news/vaccine-safety-ms/.