The month of June, just passed, was particularly rich in initiatives dedicated to some rare pathologies that mainly affect the central nervous system: on June 2nd, in fact, the World Myasthenia Gravis Day and the 23rd that of the Dravet syndrome. These are diseases for which, in recent years, UcbPharma is directing its commitment, always putting patients and their testimonies at the center, and continuing to invest in therapeutic innovations that have a positive impact on their lives.
To celebrate the two world days in the best possible way – as stated in a note – on June 24, the Belgian pharmaceutical company organized, at its offices in Milan, the event ‘Let’s relieve fatigue and leave a mark’, dedicated to employees with the aim of increasing awareness of the diseases, giving a voice to those who live with these rare diseases through the direct involvement of patient associations with video testimonials from caregivers and patients, offering participants a real and touching vision of the daily lives of these people. The initiative was attended by Isabella Brambilla, president of the Dravet Italia Association, and Roberta Annunziata, from the Aim Campania association. Following these testimonies, a creative and symbolic workshop was organized during which participants customized white t-shirts on which words representing the pain and fatigue of patients were written on the back, symbolically transferring the weight onto their shoulders. On the front, they decorated the t-shirts with zebra and unicorn stencils, symbols linked to the two diseases, leaving a tangible sign of their support and solidarity.
Myasthenia gravis (MG) – the note recalls – is a rare, chronic autoimmune disease, characterized by morphological and functional alterations responsible for the transmission disorder at the neuromuscular junction, with heterogeneous clinical manifestations such as ptosis (drooping of the eyelids), double vision, difficulty swallowing, chewing and speaking, as well as severe muscle weakness that can affect various districts and which, when involving the respiratory muscles, can be potentially lethal.
For this pathology, last December Ucb obtained European approval for zilucoplan and in January 2024 for rozanolixizumab. The first is a peptide inhibitor of complement cascade component 5 (C5 inhibitor), approved by the EU as an add-on therapy to the standard one in adult patients positive for anti-acetylcholine receptor (AChR) antibodies. It is the only drug in its category to have a dual mechanism of action. Rozanolixizumab is a humanized IgG4 monoclonal antibody, which binds to the neonatal Fc receptor, resulting in the reduction of circulating IgG. The drug is approved as an add-on therapy to the standard one in adult patients positive for AChR or anti-muscle-specific tyrosine kinase (MuSK) antibodies.
The Belgian pharmaceutical company is therefore the first and only one to have a portfolio focused on gMG, able to offer therapies targeting both anti-AChR and anti-MuSK antibodies. In May this year, the ‘Journal of Neurology’ published data from a post hoc analysis of the pivotal phase 3 Raise study and the ongoing open-label extension (Ole) Raise-XT study, which evaluates the long-term effect of zilucoplan on fatigue, in adult patients with moderate to severe gMG, positive for anti-acetylcholine receptor antibodies (AChR-Ab+).
Dravet syndrome – the note continues – is an epileptic encephalopathy that begins in childhood and is characterized by seizures that are difficult to treat with anti-seizure drugs, cognitive, behavioral and motor disorders, which persist into adulthood.
For this serious pathology, Ucb has acquired from Zogenix a drug that has been on the Italian market for about 2 years (fenfluramine), which has demonstrated a statistically and clinically significant reduction in the frequency of seizures. During the recent International Congress of Child Neurology (Icnc), the company presented the final results of the 3-year open-label extension study (OLE) of fenfluramine, which demonstrated the long-term efficacy of the drug as an add-on therapy in patients aged 2 to 32 years. Additional caregiver assessments, according to the Clinical Global Impression Scale, integrating the data on the reduction in the number of seizures, provide a positive efficacy endpoint. These results strengthen the potential of long-term treatment and the efficacy of fenfluramine up to 3 years, showing significant and durable reductions in the median monthly frequency of seizures in people with Dravet syndrome, which represent the main objectives for the patient, helping them to reach their maximum potential in development and quality of life.
“Our commitment to rare diseases – says Federico Chinni, CEO of Ucb Pharma Italia – is based on the awareness that many of these are often ‘orphan’ diseases, of limited interest to researchers and doctors, for which there is no available therapy. Choosing to undertake this path for Ucb has meant committing ourselves with courage and responsibility, to make a difference in the lives of patients with diseases that are still little known and not adequately treated. Our goal – he concludes – is to transform the life of every single person with a rare disease, developing innovative drugs, improving diagnostics and exploring new approaches to treatment”.
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