00:00Dr. Bardoscia, what has emerged from the Comet study?
00:05So, the Comet study evaluated the efficacy and safety of selumetinib,
00:10a pharmacotherapeutic, in the context of neurofibromatosis type 1 in adult patients.
00:15Selumetinib selectively inhibits MEK1 and MEK2,
00:19which are two enzymes involved in the growth of plexiform neurofibromas.
00:23The Comet study has been published in an important journal, The Lancet
00:29and is an international, randomized, double-blind, placebo-controlled, phase 3 study,
00:36which involved 145 patients, therefore a very large study, and 13 countries, including Italy.
00:43The primary endpoint of the study, which was statistically significantly met,
00:48was the objective response rate, that is, the volumetric reduction
00:53of plexiform neurofibromas measured with magnetic resonance imaging.
00:59Selumetinib also reduced pain in the study, which is a major symptom for patients,
01:05It affects almost 88% of patients.
01:09In the Comet study the reduction of this end point, therefore of pain, was approximately two points
01:16compared to the scale that was used within the study itself.
01:20Furthermore, this result was achieved both in the reduction of acute pain,
01:27which in the reduction of chronic pain, also leading to a reduction in the consumption of analgesics.
01:33Finally, the safety profile was found to be consistent with what was already known in the pediatric population.
01:40What does the expansion of selumetinib to adults mean for Alexion AstraZeneca Rare Diseases?
01:51with plexiform neurofibromas?
01:55So, for Alexion it means being able to accompany people's journeys throughout their lives.
02:01Neurofibromatosis type 1 is a rare, progressive, genetic disease
02:06often diagnosed in childhood, but which accompanies the person into adulthood
02:11and is accompanied by symptoms such as pain, deformity and functional limitations.
02:17Until now, selumetinib was only available in Italy for the pediatric population,
02:22therefore only for pediatric patients.
02:24The extension to adult patients means offering an option to those in need of treatment
02:31in this age group, but it also means making the transition of care smoother
02:38from childhood to adulthood, which is often a delicate transition in all
02:45pathologies, but particularly rare diseases.
02:48Furthermore, this approval is undoubtedly not a point of arrival for Alexion,
02:54but a starting point, in fact Alexion's goal is to continue to generate
03:00evidence that supports the data from the randomized clinical trial also in practice
03:08Italian clinic, where there was a long-term collection of real-life data
03:13which confirmed the efficacy and safety of selumetinib observed in clinical studies.
03:19What is the company's commitment to rare genetic diseases?
03:26For over 30 years Alexion AstraZeneca Rare Disease has been working in rare diseases with a principle
03:33clear and precise, which is to understand the diseases, the treatment path of patients
03:39and generate solid evidence to then translate it into concrete therapeutic opportunities.
03:47Alexion's commitment goes beyond the drug, why?
03:53Because it means listening to the ecosystem in all its actors, namely the associations
03:58patients, scientific societies and institutions, in order to support disease activities
04:05awareness and training projects that can help recognize warning signs
04:12of the disease and to direct the person towards appropriate and virtuous diagnostic paths.
04:19The aim of all these actions is to reduce the diagnostic delay, which in
04:24Rare diseases still represent a real challenge today.
04:28Therefore our ambition is to build increasingly continuous care pathways, which
04:34ranging from diagnosis to treatment, in which scientific innovation can be translated
04:41into concrete answers for people living with rare diseases and for their families.
