March 27, 2019 News by Jose Marques Lopes, PhD Celgene Seeking FDA Approval for Ozanimod to Treat Adults With Relapsing MS An application has been submitted to approveĀ ozanimod as an oral treatment for adults with relapsing forms of multiple sclerosis in the U.S., according to its developer,Ā Celgene. āNew oral treatment options with differentiated profiles like ozanimod are needed to help address an unmet need for people with relapsing forms of MS,ā Jay Backstrom, MD, Celgeneās chief medical officer, said in a press release. Celgene'sĀ New Drug Application has been submitted to the U.S. Food and Drug Administration. Earlier this month, the companyĀ submitted a marketing authorization application to the European Medicines Agency covering the treatment of adults with relapsing-remitting MS. āWith concurrent applications in the U.S. and EU, we look forward to advancing this promising medicine through the regulatory review process to provide a new option for the treatment of (relapsing MS) in 2020,ā Backstrom said. Ozanimod is designed to cause the retention of immune cells in lymphoid tissues, thereby blocking their migration to the central nervous system ā brain and spinal cord ā and preventing damage to nerve fibers and their protective layer, called myelin. The investigational therapy selectively binds to S1P receptor subtypes S1P1 and S1P5. The NDA application is based on positive findings from two multicenter, double-blind, Phase 3 trials calledĀ SUNBEAM andĀ RADIANCE part B. Both studies demonstrated that ozanimod reduced the number of relapses and brain lesions. In theĀ SUNBEAMĀ Phase 3 trial, 1,346 participants with relapsing MSĀ were randomized to one daily dose of 0.92 or 0.46 mg of ozanimod ā equivalent to 1 mg and 0.5 mg of the therapyās HCI formulation ā orĀ AvonexĀ (interferon beta-1a, marketed byĀ Biogen) for at least 12 months. Results showed that treatment with ozanimod led toĀ fewer relapsesĀ and brain lesions, as well as clinically meaningful improvements in processing speed compared with Avonex. In the Phase 2/3 RADIANCE trial, patients were divided in two parts: in part A, participants received either one daily dose of ozanimod (0.5 mg or 1.0 mg) or a placebo for 24 weeks; in part B, a 96-week open-label extension study completed by 223 patients, those initially on placebo switched to ozanimod. As in the SUNBEAM trial, results of part A of the RADIANCE trial revealed a reduction in the number of brain lesions from weeks 12 to 24, as well as less frequent relapses compared with a placebo. Treatment with ozanimod was safe and well-tolerated. Findings of part B of the studyĀ included an increased percentage of patients free of T1 lesions on MRI (magnetic resonance imaging) scans ā which refer to areas of active inflammation and disease activity ā after two years of treatment, from 58.5ā69.0% of patients in part A to 86.5ā94.6% of patients in part B. T2 lesions, a measure of the total amount of MRI lesions ā both old and new ā and relapse rate remained low in patients maintained on ozanimod (more significantly with the higher dose of 1.0 mg), and dropped in those who switched from a placebo. The scientists also analyzed ozanimodās benefits usingĀ data from the SUNBEAM and RADIANCE part B trials, which covered 2,659 patients treated over one to two years. Compared with Avonex, ozanimod reduced the annualized relapse rates ā the number of relapses per year ā by 42% in the higher dose group and 26% in the lower dose group. Treatment with ozanimod also lessened the relapse rate requiring steroid treatment or hospitalization by 43% (in the 1 mg dose group) and 26% (in the 0.5 mg dose group) compared with Avonex treatment. In addition to MS, ozanimod is also being developed for patients with ulcerative colitis and Crohn's disease, two inflammatory bowel diseases.
March 27, 2019 News by Patricia Inacio, PhD FDA Approves Novartis’ Mayzent for Relapsing Forms of Multiple Sclerosis, Including Active SPMS The U.S. Food and Drug Administration (FDA) has approved Novartis’ Mayzent (siponimod) oral tablets for adults with relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome (CIS), relapsing-remitting disease (RRMS), and active secondary progressive disease (SPMS). Mayzent was designed to inhibit the activity of…
March 12, 2019 News by Jonathan Grinstein Celgene Seeks Approval in European Union for Ozanimod to Treat Adult RRMS An application has been submitted for the use of ozanimod to treat adult patients withĀ relapsing-remitting multiple sclerosis (RRMS)Ā within the European Union, according toĀ Celgene, the developer of ozanimod. The marketing authorization application was submitted to the European Medicines Agency…
February 27, 2019 News by Alice MelĆ£o, MSc FDA Will Review New Drug Application of Diroximel Fumarate for Relapsing Forms of MS The U.S. Food and Drug Administration (FDA) has agreed to review Alkermesā request to approve diroximel fumarate (BIIB098) as a treatment for relapsing forms of multiple sclerosis (MS), the company announced. A final decision by the FDA is expected in the fourth quarter of 2019. If approved, diroximel…
February 21, 2019 News by Vijaya Iyer, PhD FDA Warns Against Plasma Transfusions from Young Donors Being Used to Treat MS and Other Diseases The U.S. Food and Drug Administration (FDA) has warned against the use of plasma transfusions from young donors toĀ alleviate or treat the symptoms ofĀ multiple sclerosisĀ or other diseases, noting such transfusionsĀ have no proven clinical benefitĀ and carry known health risks associated with their use. Plasma is the liquid component of blood, containing proteins that help in clotting, and can be used to treat bleeding disorders and cases of trauma. But its use inĀ transfusions as a means of treating conditions ranging from multiple sclerosis, dementia, Alzheimerās,Ā Parkinsonās, heart disease and post-traumatic stress disorder are of concern, the FDA said in issuing itsĀ statement of Feb 19. "We have significant public health concerns about the promotion and use of plasma for these purposes," the FDA statement reads. "[W]eāre alerting consumers and health care providers that treatments using plasma from young donors have not gone through the rigorous testing ā¦ [necessary] to confirm the therapeutic benefit of a product and to ensure its safety." Scott Gottlieb, theĀ FDA's commissioner, and Peter Marks, director of its Center for Biologics Evaluation and Research, jointly issued the statement cautioning healthcare providers and the public that plasma infusions ā being done at "a growing number of clinics" in several U.S. states ā are not an FDA-approved or recognized treatment for aging, memory loss, multiple sclerosis, or other diseases. FDA-approved treatments largely come through clinical trials overseen by researchers and independent boards, and performed under anĀ investigational new drug (IND) applicationĀ that helps to ensure patient safety. āOur concerns regarding treatments using plasma from young donors are heightened by the fact that there is no compelling clinical evidence on its efficacy, nor is there information on appropriate dosing for treatment of the conditions for which these products are being advertised,ā theĀ statement notes. According to the FDA, large volumes of plasma might be also be needed for such transfusions and that volume can pose significant risks, including allergic reactions, infections, and heart and respiratory problems. The agency also expressed concern that such transfusions could discourage patients from taking medications or other treatments known to be safe and effective for their condition. According to the agency, some "establishments" across the country are recommendingĀ young donor plasma infusions and "touting" them "as cures and remedies," while sometimes "charging thousands of dollars." The FDA advises patients to consult with their treating physician before opting for any treatment to confirm that it is approved for use, meaning its safety and effectiveness have been demonstrated. āSimply put, weāre concerned that some patients are being preyed upon by unscrupulous actors touting treatments of plasma from young donors,ā the FDA statement reads. It also asks that patients who have undergone a plasma transfusion report any ill effects to its MedWatch program, which tracks adverse events related to treatments.
February 14, 2019 News by Jonathan Grinstein FDA Asked to Place Cannabis-based Therapy for Progressive MS on Fast Track, MMJ Holdings Says A request for a potentialĀ cannabis-based treatmentĀ forĀ multiple sclerosisĀ to be givenĀ Fast Track designation, speeding its development as it readies to enter clinical testing, is now before theĀ U.S. Food and Drug Administration (FDA), MMJ International HoldingsĀ announced. MMJ-001, as this lead candidate is known, aims to treat…
January 4, 2019 News by Alberto Molano, PhD Bafiertam, a Bioequivalent of Tecfidera, Receives Tentative OK from FDA for RRMS Banner Life SciencesĀ has received tentative approval from the U.S. Food and Drug Administration (FDA) for its new drug application (NDA) for Bafiertam (monomethyl fumarate), a novel bioequivalent of Biogenās Tecfidera (dimethyl fumarate) for the treatment of relapsing-remitting multiple sclerosis (RRMS). Final approval is expected no later than…
December 19, 2018 News by Alberto Molano, PhD FDA Gives Green Light to Phase 2 Trial of NurOwn Stem Cell Therapy in Progressive MS Patients The U.S. Food and Drug Administration (FDA) has approvedĀ BrainStorm Cell Therapeutics‘ request to open a Phase 2 clinical trial testing the safety and effectiveness of its proprietary NurOwn mesenchymal stem cell (MSC)Ā treatment inĀ progressive multiple sclerosis (MS) patients. The request was in the form of Investigational New Drug…
December 18, 2018 News by Alice MelĆ£o, MSc FDA Asked to Approve Diroximel Fumarate as Oral Treatment for Relapsing MS AlkermesĀ filed a request for the approval of diroximel fumarate (BIIB098) to treatĀ relapsing forms of multiple sclerosis (MS) with the U.S. Food and Drug Administration (FDA). If approved, diroximel fumarate will be marketed by BiogenĀ in the U.S., likely under the brand name Vumerity. Alkermes and Biogen are working…
December 3, 2018 News by Ana Pena PhD Lemtrada’s Use Carries Rare But Serious Risk of Stroke and Artery Tears, FDA Warns The U.S. Food and Drug Administration (FDA)Ā has issued a safety alert, warning about a rare but life-threatening risk of stroke and artery rupture in patients with relapsing forms of multiple sclerosis (MS)Ā being treated with Lemtrada (alemtuzumab). Since Lemtrada’s approval in 2014 to treat relapsing MS, 13…
November 27, 2018 Columns by Ed Tobias FDA Warns of Possible Dangers of Stopping Gilenya If you are being treated with Gilenya, take note. The U.S. Food and Drug Administration is warning that if you stop using Gilenya (fingolimod), there’s a chance your MS could become worse. The FDA issued a safety alert saying that this only happens rarely, but when it does, the…
November 26, 2018 News by Ana Pena PhD FDA Warns of Rare Risk of Severe Worsening in MS Disability After Stopping Gilenya Use The U.S. Food and Drug Administration (FDA) has issued a safety alert, warningĀ that people with relapsingĀ multiple sclerosis (MS)Ā who stop usingĀ Gilenya (fingolimod) may experience disease worsening beyond that when starting the medicine or while taking it. Reported cases of such increases in MS disability upon stopping treatment are…
November 21, 2018 News by Alice MelĆ£o, MSc Phase 2 Trial of NurOwn Stem Cell Therapy in Progressive MS Planned for US, BrainStorm Announces BrainStorm Cell Therapeutics is planning to launch a Phase 2 clinical trial in the United States to evaluate the safety and activity of its lead cell therapy candidate,Ā NurOwn, in people with progressive multiple sclerosis (MS). The company announced that has submitted an Investigational New Drug (IND) application to…
November 20, 2018 News by Alice MelĆ£o, MSc FDA Approves Generic Version of Aubagio to Treat Relapsing MS, Glenmark Pharma Says The U.S. Food and Drug Administration (FDA) has approved a generic version of Ā Aubagio (teriflunomide) tablets at the 7 mg and 14 mg doses marketed by Sanofi, according to the generic’s manufacturer,Ā Glenmark Pharmaceuticals. The FDAās decision to approve the company’s application for teriflunomide tablets at two…
October 15, 2018 News by BioNews Staff #ECTRIMS2018 – Post-hoc Analyses Support Safety and Efficacy of Mavenclad, Merck KGaA Says Substantial data supporting both the effectiveness and safety of Mavenclad (cladribine tablets) is before the U.S. Food and Drug Administration (FDA), and may lead to its approval as a short-course oral treatment for people with relapsing-remitting multiple sclerosis (RRMS) some seven years after a first such request…
October 10, 2018 News by Alice MelĆ£o, MSc FDA, EMA Agree to Review Novartis Therapy Siponimod to Treat SPMS Novartis is seeking U.S. and European approval of its investigational oral agent siponimodĀ to treat adults with secondary progressive multiple sclerosis (SPMS). The U.S. Food and Drug Administration (FDA) has accepted for review the company’s New Drug Application, while the European Medicines Agency (EMA) has accepted for review…
September 6, 2018 Columns by Laura Kolaczkowski Stem Cell Therapy and Circumvention Tourism Medical tourism is a term describing when people seek medical care by traveling from home countries to somewhere else. Itās an area of commerce that has existed for centuries, as people in ancient Greece once traveled to far away islands to visit healing gods. Medical tourism continues today, and…
August 28, 2018 News by Vijaya Iyer, PhD MMJ International Asks FDA to Approve Studies of Cannabis-based Therapies MMJ International has filed two applications with the U.S. Food and Drug Administration (FDA) requesting permission to begin clinical studies testing its pharmaceutical grade cannabis-based therapies in easing symptoms associated with multiple sclerosis and Huntingtonās disease. āThe filing of these applications with the FDA brings us one step…
July 31, 2018 News by Alice MelĆ£o, MSc FDA to Review EMD Serono’s New Request for Approval of Cladribine for Treating Relapsing MS Seven years after it first denied the request, the U.S. Food and Drug Administration accepted for review EMD Seronoās resubmitted New Drug Application (NDA) for cladribine tabletsĀ (brand name Mavenclad) as a treatment for patients with relapsing forms of multiple sclerosis (MS). The regulatory agency agreed EMD…
June 13, 2018 News by Patricia Silva, PhD MMJ Bioscience Files Request with FDA to Open Phase 2 Study of Medical Cannabis in Progressive MS MMJ Bioscience, which specializes in medical cannabis products, has filed a request with the U.S. Food and Drug Administration (FDA) to open Phase 2 clinical trials evaluating the companyās THC/CBD pharmaceutical compounds as possible treatments for symptoms ofĀ multiple sclerosis (MS). THC refers to the tetrahydrocannabinol compound, part of the…
May 25, 2018 Columns by Ed Tobias Two MS Medications Appear on FDA’s Shame List Two multiple sclerosis medications that are both popular and expensive are on a new list that U.S. regulators hope will increase price competition. The medications are Ampyra (dalfampridine), made by Acorda, and TecfideraĀ (dimethyl fumarate), by Biogen. They’re included on a list of more than 50…
May 23, 2018 News by Diogo Pinto European EMA Confirms Severe Risks Associated with MS Medicine Zinbryta A review of data concerning the multiple sclerosis (MS) medicine Zinbryta (daclizumab) confirmed its association with the risk of developing severe and potentially fatal immune reactions in the brain, liver and other organs, according to the European Medicines Agency’s (EMA)ās Pharmacovigilance Risk Assessment Committee (PRAC). Zinbryta was authorized…
May 15, 2018 News by Alice MelĆ£o, MSc Gilenya Approved by FDA as First DMT for Children, Ages 10 and Older, with Relapsing MS Gilenya (fingolimod) has become the first disease-modifying therapy approved by the U.S. Food and Drug Administration (FDA) to treat children and adolescents with relapsing forms of multiple sclerosis (MS). This expanded approval allows Gilenya, previously indicated for adults patients 18 or older, to be used to treat pediatric relapsing MS…
April 13, 2018 News by Patricia Inacio, PhD Acute Acalculous Cholecystitis Linked to Lemtrada Use in RRMS Patients, FDA Reports Acute acalculous cholecystitis (AAC) is a rare but potentially life-threatening adverse effect linked to treatment with Lemtrada (alemtuzumab)Ā in patients with relapsing-remitting multiple sclerosis (RRMS), according to a U.S. Food and Drug Administration review. The study, āAcute acalculous cholecystitis ā A new safety risk for…
April 2, 2018 News by Patricia Silva, PhD FDA Clears Mobile App for Use with L300 Go System Created to Help with Walking and Gait The U.S. Food and Drug Administration (FDA) recently cleared the myBioness mobile app, designed by Bioness, to be used with the L300 Go SystemĀ to improve muscle strength in patients with multiple sclerosis (MS) or other conditions that affect walking. The L300 Go System is approvedĀ to help…
March 14, 2018 News by Jose Marques Lopes, PhD MMJ BioScience Obtains First Canadian License to Produce Medical Cannabis MMJ Bio ScienceĀ has becomeĀ the first company to obtain a Canadian license to produce medical cannabis, with the initial authorization covering products forĀ multiple sclerosisĀ andĀ Huntington’s disease. The Health CanadaĀ license allows the company to extract cannabinoids from plants that it grows to produce therapies that will be tested in U.S.
March 2, 2018 News by Alice MelĆ£o, MSc FDA Wants More Information on Celgene’s MS Therapy Ozanimod Before Starting Its Review The U.S. Food and Drug Administration has asked CelgeneĀ to add more information to its New Drug Application for ozanimod as a treatment for relapsing forms of multiple sclerosis. Celgene said it plans to meet with FDA officials to make sure it understands exactly what new information the agency…
February 15, 2018 News by Patricia Silva, PhD FDA Approves Dose of MS Therapy Glatopa That Is Twice as Large as Current One The U.S. Food and Drug Administration has approved a new dose ofĀ Sandozās multiple sclerosis therapyĀ Glatopa (glatiramer acetate injection)Ā that is twice as large as the currently authorized one. Regulators’ approval of theĀ 40 mg/mL applies to people with relapsing forms of MS. A mg/mL designation refers to the concentration of…
January 30, 2018 News by Patricia Silva, PhD FDA Approves Swedish Company’s Technology for Getting More out of MRI Scans The U.S. Food and Drug Administration has approved technology that the Swedish companyĀ SyntheticMRĀ developed to give doctors more information from magnetic resonance imaging scans. This means the company can begin selling itsĀ SyMRI NEUROĀ packages to American medical facilities. Traditional MRIs offer only one level of contrast when depicting tissue.Ā SyMRI NEURO…
January 9, 2018 News by Alice MelĆ£o, MSc American CyroStem Warned by FDA to Stop Using Stem Cell Therapy Without Approval and to Correct Safety Issues American CryoStem has received a warning letter from the U.S. Food and Drug Administration (FDA) for marketing its adipose-derived stem cell product Atcell without required regulatory approval, and for "significant deviations" from manufacturing processes that potentially raise safety concerns. The company has 15 working days to respond to the concerns raised by the agency and detail how they will be corrected, or risk "enforcement actions," the FDA said in a Jan. 4 press release. StudiesĀ suggest that mesenchymal stem cells can be used to alleviate symptoms and possibly treat several degenerative disorders, including multiple sclerosis. Atcell is aĀ therapy based on the ability of mesenchymal stem cells isolated from adipose tissue (fatty tissue) to transform into a subset of mature cells, which include adipose cells, bone cells, and cartilage cells. Although not approved for use, Atcell is being distributed directly to physicians to treat patients affected by several life-threatening diseases, including Parkinsonās disease, amyotrophic lateral sclerosis, stroke, and multiple sclerosis, the FDA said in its release. It is administeredĀ intravenously, intrathecally (injection or infusion into the central nervous system) or by aerosol inhalation. The productĀ is designed to be used in the same individual (autologous use) the cells are collected from, an approach intended to reduce risk. The cells are extractedĀ using the companyās proprietary Cellect collection system. They are then expanded in the laboratory using the company's ASCelerate SF-M serum free (animal-product free) media,Ā providing compounds needed for the cells to survive and proliferate.Ā Stem cells put through this process are Ā ready to be used as therapy or to be stored for future use. A recent FDA inspection found that Atcellās manufacturing steps are not in line withĀ current good manufacturing practice requirements. Specifically, the manipulation of the adipose tissue was more than "minimal," the FDA reported, changing "relevant characteristics" of the original tissue that could introduce contamination by microorganisms or product defects and represent a "risk of harm" to patients. Because of this manipulation, FDA review is required by law to ensure Atcell's safety and efficacy, the agency said. Evidence of an inadequately controlled manipulation environment, lack of control of components used in production, andĀ insufficient and inadequately validated product testing were also reported. āThe use of Atcell raises potential significant safety concerns, due in part to the fact that there is little basis on which to predict how the product will perform in a patient,ā Peter Marks, director of the FDAās Center for Biologics Evaluation and Research, said in the release. āIn addition, this product may also cause harm to patients who may put their trust in an unproven therapy and make the decision to delay or discontinue medical treatments proven to be safe and effective,ā Marks added. American CyroStem, based in New Jersey, did respond to observations raised by the FDA at the time of its inspection. But they were found inadequate to support Atcellās marketing, and failed to acknowledge that FDA approval was required, either by filing a biological license or investigational new drug application. āAs part of our comprehensive policy framework for the efficient development and regulation of cell-based regenerative medicine, weāre going to be stepping up enforcement activities against those who manufacture and market products in ways that put patients at risk,ā said Scott Gottlieb, FDA commissioner. āWe see great promise from the field of cell based regenerative medicine, but there are also novel risks,ā Gottlieb added. Healthcare professionals and patients who have used Atcell are asked to report any adverse events related to the treatment using the FDAāsĀ MedWatch Online Voluntary Reporting Form. Completed forms can be submitted online, or via fax to 1-800-FDA-0178. Ā