FDA Accepts Application for Cladribine Tablets

EMD Serono, Inc. announced today that the United States Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for Cladribine Tablets (an oral formulation of cladribine), an investigational treatment for relapsing forms of MS. Accepted by the European Commission in August 2017 and by Canada in December 2017, Cladribine is currently approved for the treatment of relapsing MS in 38 countries under the trade name Mavenclad®.

In studies, cladribine has been shown to reduce disease activity in patients with relapsing MS, including disability progression, annualized relapse rate, and MRI activity. Potential adverse events include lymphopenia, a condition that causes abnormally low counts of white blood cells that play a role in fighting infection, and herpes zoster infection.

According to MSAA’s MS Research Update 2018, “Cladribine selectively targets the immune system’s B cells and T cells, leading to depletion of those cells. This is followed by a distinct pattern of ‘reconstitution,’ as new B cells and T cells are produced. The medication has an interesting dosing regimen, with two annual courses given for a maximum of 20 days over two years. Its developers note that this approach avoids continuous suppression of the immune system.” Following these first two years, no treatment is needed for Years 3 and 4.

In a press release issued today, EMD Serono states, “The NDA acceptance includes close to 12,000 patient years of data with over 2,700 patients included in the clinical trial program, and up to 10 years of safety data in some patients. The clinical development program included data from three Phase III trials, CLARITY, CLARITY EXT and ORACLE MS, the Phase II ONWARD study and long-term follow-up data from the eight-year prospective registry, PREMIERE.”

An application for approval by the FDA was submitted and received Fast Track status in July 2010. However, in March 2011, the pharmaceutical manufacturer received a complete response letter (CRL) from the FDA, stating that they could not approve the application without additional information. While the effectiveness was not in question, the FDA was looking for an improved understanding of safety risks and the overall benefit-risk profile. This required either additional analysis of available data, or additional studies to be conducted. For more information, please see MSAA’s article, “Oral Cladribine Denied FDA Approval.”

MSAA Chief Medical Consultant Dr. Jack Burks explains, “The experimental medication cladribine has been studied as a long-term treatment for relapsing forms of MS for many years. Already approved in 38 countries, we are very happy to learn that it is now under review by the FDA for possible approval here in the United States. The potential addition of another disease-modifying therapy for the MS community is always exciting, as it presents a greater opportunity for patients and their healthcare team to select a treatment plan that best fits their needs. We look forward to hearing back from the FDA in the months ahead.”

For general information or to speak with a trained Client Services Specialist, please call MSAA’s Helpline at (800) 532-7667, extension 154. Questions to MSAA’s Client Services department may also be emailed to MSquestions@mymsaa.org.

Written by Susan Wells Courtney, MSAA Senior Writer

Reviewed by Dr. Jack Burks, MSAA Chief Medical Consultant