The first drug for home therapy of patients with spinal muscular atrophy (SMA) arrives in Italy in its most widespread forms, types 1, 2 and 3. Aifa has approved the marketing and reimbursement of risdiplam, “an innovative solution studied on a large and heterogeneous sample of the population, both in terms of age and pathology characteristics – underlines Roche announcing the launch of the treatment – which has proved effective in all target patients studied. “The administration of the medicine, a strawberry flavored syrup, is daily and takes place orally.
“Risdiplam is a small molecule that allows to restore the production of functional Smn proteinand “, the one missing in SMA patients, who undergo motor neuron degeneration with muscle atrophy and paralysis,”thus reducing the symptoms of the disease and slowing its progression – explains Eugenio Mercuri, Director of Child Neuropsychiatry A. Gemelli Irccs University Polyclinic Foundation, Catholic University of Rome – The efficacy and safety of this new therapeutic option are proven by the vast program of clinical studies that involved a very large patient population, both by age and by features of pathology. These include the two pivotal trials Firefish and Sunfish, and the Jewelfish studies, in patients who have received previous therapy, and Rainbowfish in presymptomatic patients “.
“Risdiplam represents the first home therapy for SMA with proven efficacyconfirming Riccardo MassonMedical Director Child Neuropsychiatry, Developmental Neurology Irccs Foundation Carlo Besta Neurological Institute of Milan – which constitutes a potential transformation in the way a large population of people living with the disease is treated. By avoiding the need for administration in a hospital setting, as well as facilitating the life of patients and their caregivers, risdiplam can promote adherence to the therapeutic protocol. To support the home management of the drug, doctors will also have at their disposal a package of services aimed at facilitating the monitoring and progress of the treatment path “.
The service package – a note details – provides that patients receive, in addition to the home delivery of risdiplam, a refrigerated bag useful for maintaining the cold chain until the drug is delivered at home, and a scale for patients under 20 kilograms so that can weigh themselves directly at home and facilitate the clinician in monitoring any dose adjustments, which in patients under 20 kg is weight dependent. The innovation of this package also concerns the monitoring of the patient by the clinician, who, thanks to an App, can control and manage all aspects of therapy at a distance.za, monitoring daily adherence and other elements that may impact the drug profile.
“The arrival of a new therapy is always a great joy for our community – he comments Anita Pallarapresident of Sma Families – The first oral administration marks another, epochal, step forward. Risdiplam is an important weapon to combat the disease, thanks to which we will reach an increasing number of people. We thank the authorities and the company for the commitment and dedication with which they have made this new opportunity concrete in the shortest possible time. We are aware that it is a new challenge, ready to face it “.
“The search for therapeutic solutions and innovative services capable of responding to the needs of patients and those close to them is in Roche’s DNA, and risdiplam is one of the fruits of this constant commitment – he declares Anna Maria PorriniRoche Italia Medical Director – Risdiplam received prestigious awards, including the Prix Galien 2021 innovation award in Italy for the ‘Orphan Drugs’ category. We are really proud to be able to make the first home treatment available in Italy for the most common types of SMAproviding a new therapeutic solution for people who live with this pathology on a daily basis “.
Risdiplam is already available in our country from 2020 thanks to the compassionate use program for patients with type 1 and type 2 SMA, recalls Roche. The go-ahead from the Italian Medicines Agency now marks “an important turning point in the panorama of rare diseases and in the management of the disease – the company remarks – which for the first time can be treated directly at home, in a historical moment in which a cause of the Covid-19 pandemic, we want to minimize access to hospital for the most vulnerable people, favoring therapeutic paths that can be managed independently by the patient and his caregiver “.
The overall incidence of all forms of SMA is estimated in one case out of 7-10 thousand live births, the note continues. The prevalence of SMA types 2 and 3 is estimated to be between 40 cases per million children and 12 cases per million in the general population. To date, over 4,500 patients worldwide have had access to risdiplam therapy.
In Italy, 85% of the centers that deal with the treatment of SMA patients have already been able to use the treatment with risdiplam. Of the 1,300 estimated SMAs in our country, more than 300 have already been treated with risdiplam in clinical trials, compassionate use programs, and thanks to an early access program. The tolerability of the therapy was very positive; there were no discontinuation situations in clinical trials for safety reasons.
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