Multiple Sclerosis: New Therapeutic Options for Patients. Green light from AIFA for Mayzent® (siponimod)
e or EMA per Kespta® (ofatumumab)
Two good news for people with multiple sclerosis (MS). With the Official Gazette no. 88 AIFA has given the green light to the reimbursement of siponimod, the first oral therapy indicated for secondary progressive multiple sclerosis (SPMS) with disease with signs of activity. This result comes shortly after the EMA approval of another multiple sclerosis therapy: ofatumumab, a B-cell targeted treatment indicated for adult patients with relapsing multiple sclerosis (RRMS). Two important approvals that testify to Novartis’ commitment to developing therapies capable of responding to the still unmet needs of people with MS in different forms of disease and stage of disability.
Multiple sclerosis is a chronic disease that affects the central nervous system and can cause disruption of signals between the brain, spinal cord, and optic nerves, leading to a wide range of symptoms. According to the largest global survey on the disease, published by the International Multiple Sclerosis Federation (MSIF), neurological diseases, of which multiple sclerosis is a part, are among the most impacting in the Italian population: they are positioned as the third cause of death. after cardiovascular pathologies and tumors, and the third cause of disability.
According to the MS Barometer in Italy drawn up by the Italian Multiple Sclerosis Association, in Italy over 126 thousand people in Italy are forced to live with the chronic course of the disease. For this reason, the goal is to undertake an adequate and specific therapeutic path for one’s form of MS to slow down its progression and reduce disability, in order to improve one’s quality of life.
Not only that, the slowdown in the progression of the disease and the reduction of the associated disability also represent a relief from the socio-economic burden of MS today quantified on average at 45,000 euros per year per person with MS which can reach the figure of 84,000 euros in case advanced multiple sclerosis.
SIPONIMOD: AN ORAL TREATMENT TO DELAY THE PROGRESSION OF DISABILITY AND OBTAIN COGNITIVE BENEFITS
The Phase III EXPAND trial is the largest randomized controlled trial conducted to date in SPMS and has demonstrated the efficacy and safety profile of siponimod. The results obtained confirmed that the risk of confirmed disability progression (CDP) at three and six months, in patients with disease with signs of activity, was significantly reduced by 31% and 37%, respectively, in the group treated with siponimod in comparison. to placebo. Significant findings were also noted regarding the annualized relapse rate (ARR), MRI disease activity, and brain volume loss. Further exploratory analyzes have shown that siponimod can help patients delay wheelchair use by more than four years on average and also determines an important benefit also on cognitive functions by demonstrating clinically relevant effects on the speed of cognitive processing evaluated through the Symbol Digit. Modalities Test (SDMT).
“This is possible because siponimod, thanks to its mechanism of action, has a double efficacy, acting both on the inflammation coming from the periphery and mainly driven by lymphocytes, and on the compartmentalized central inflammation that is established within the central nervous system and is one of the main drivers of secondary progression in MS – explained Dr. Luigi Lavorgna, Neurologist AOU “Luigi Vanvitelli” University, Chair of the “Digital” Study Group of the Italian Society of Neurology – We are facing an innovative treatment that gives us hope in an improvement both in the care and in the quality of life of people with MS for whom it is extremely important to delay the progression of disability and who until now did not have an effective therapy “.
OFATUMUMAB: A TARGETED, EFFECTIVE AND SELF-ADMINISTRATED THERAPY
Ofatumumab is the first targeted B-cell therapy that has demonstrated superior efficacy and a similar safety profile to teriflunomide, one of the first-line treatments for MS to date. The two twin Phase III studies ASCLEPIOS I and II, on which the EU approval is based, demonstrated a reduction in annual relapses of more than 50% compared to teriflunomide and a reduction in the relative risk of disability progression over 3 months. to 30%. Ofatumumab can also be self-administered once a month directly at home, and is therefore a candidate to become a first choice treatment option for patients with relapsing forms of multiple sclerosis (RMS). After EMA approval, which follows that of FDA in the United States, AIFA is expected to make the treatment available for
Italian patients.
“The efficacy coupled with the flexibility and ease of administration of ofatumumab make it a potential first choice treatment to help improve the quality of life of people living with MS. Facilitating their access to therapy can lead to benefits. both in the short and long term as well as having an impact in potentially reducing the medical costs associated with infusion therapies – commented Professor Marinella Clerico, Head of SD Specialized Neurological Pathologies, AOU San Luigi Gonzaga and Associate Professor, Department of Clinical and Biological Sciences UniTo – Slowing down the worsening of disability is one of the main goals in managing MS and our data show that early initiation of highly effective treatment can have long-term positive outcomes both on the patient’s quality of life and in terms of health costs related to disability “.
TWO IMPORTANT APPROVALS FOR UNSATISFIED NEEDS
“The unsatisfied needs of people with multiple sclerosis concern comprehensive and personalized care, with difficulties in accessing therapies, rehabilitation and aids, but also lack of answers to needs of a socio-health and social nature, such as work, transport, social participation especially in this particular historical moment – commented Francesco Vacca, National President of AISM Onlus – The availability of new specific therapies for the different forms of disease, which are framed in the scenario of the already existing therapeutic options, therefore has the potential to reduce the significant physical, emotional and financial burden that MS places on people and their families, improving their expectations and quality of life and new prospects for care are the significant result of the great commitment that the community of clinicians and researchers dedicates, together with people with multiple sclerosis, to fight the disease “.
“MS is a complex disease that can significantly affect the autonomy of patients and caregivers. It is important to have a range of treatments with different mechanisms of action and routes of administration, capable of responding to the needs of the different forms of disease – stated Gaia Panina, Chief Scientific Officer Novartis Italia – Novartis’ commitment to patients with MS is to reimagine medicine to improve the lives of patients who are affected by it, supporting innovation and investing in new solutions, not only pharmacological, capable of accompanying and supporting clinicians and patients in the diagnostic-therapeutic path “
Information about siponimod
Siponimod is a sphingosine-1-phosphate receptor modulator that selectively binds to S1P1 and S1P5 receptors. In relation to the S1P1 receptor, it prevents lymphocytes from exiting the lymph nodes and, consequently, from entering the central nervous system (CNS) of patients with MS6. This results in the anti-inflammatory effect of siponimod. Siponimod also penetrates the CNS and binds to the S1P5 sub-receptor on specific cells, including astrocytes and oligodendrocytes, and has been shown to exert remyelinating and neuroprotective effects in preclinical models of MS.