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.
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