(Adnkronos) - L’Agenzia italiana del farmaco (Aifa) ha approvato la rimborsabilità di asfotase alfa, una terapia enzimatica sostitutiva per il trattamento di pazienti affetti da ipofosfatasia (Hpp) ad esordio pediatrico. La terapia, messa a punto da AstraZeneca e Alexion, AstraZeneca Rare Disease è la prima terapia per l'Hpp, disordine metabolico sistemico raro caratterizzato da un'alterazione nella struttura ossea determinata dall'assenza di un enzima, la fosfatasi alcalina.
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00:00The IFA, the Italian pharmaceutical agency, has approved the reimbursement of Asphotase
00:09Alpha, a long-term substitutive enzymatic therapy for the treatment of patients affected
00:14by hypophosphatase at pediatric discharge, a rare disease that in severe forms affects
00:20a person out of 300,000 in the general population.
00:23To announce it AstraZeneca, together with Alexion AstraZeneca Rare Disease, with a meeting
00:29with the press in Milan, the hypophosphatase or HPP in children involves premature loss
00:35of deciduous teeth, skeletal deformities, delay in walking and rachitism.
00:40Children who have a great difficulty in the deambulation, large physical disproportions,
00:47obvious physical anomalies, which after the start of therapy certainly have an improvement
00:54in physical performance, but the most striking part is at the radiographic level, a bone
01:01seriously disorganized, with a cartilage of growth, with signs compatible with an
01:07important rachitism, I do not say that it is completely restored, but there is a clear
01:14improvement, both in bone mineralization and in the formation of new bone tissue.
01:22In the adult, hypophosphatase manifests itself with fractures, pseudo-fractures, pain in
01:29skeletal muscle, fatigue, dental anomalies, difficulty in deambulation.
01:34Italy did not have a great experience, simply because the drug was not refundable, so we
01:41could only give it to the most serious patients.
01:47There was a patient in particular who was now lying in bed, with femoral fractures,
01:53multiple vertebrae, assisted by the family.
01:56After the pharmacological treatment, she came to my studio with a single stick, which
02:01for me was a miracle.
02:03The results observed with alpha-asphotase are encouraging and improvements are noticeable
02:08in a few weeks of supply.
02:10The treatment is a beacon for patients.
02:14Finally we have the drug for children and young adults, which will change their lives
02:20in a total way.
02:22For over 30 years, Alexion has been committed to the research for the development of innovative
02:27therapies for people affected by rare diseases.
02:29For about 15 years we have started the development of a substitutional enzymatic therapy for
02:36alpha-asphotase, for the treatment of hypophosphatase.
02:41We have taken risks in the field of research, for example in hypophosphatase, and today we
02:50can be proud to say that this risk is worth taking, because we have been able to provide
02:58patients affected by hypophosphatase with this important therapeutic innovation.