GSK’s Strimvelis gains approval as gene therapy for kids with rare disorder
GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) announced has been approved by the European Commission as the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency).
A child born with ADA-SCID does not have a healthy, fully-functioning immune system and as a consequence, is unable to fight off everyday infections. Strimvelis (autologous CD34+ cells transduced to express ADA) is the first corrective gene therapy for children to be awarded regulatory approval anywhere in the world. It is indicated for the treatment of patients with ADA-SCID for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
ADA-SCID affects an estimated 15 children per year in Europe and following the approval, patients with the condition who are referred for treatment will be able to receive the gene therapy at Ospedale San Raffaele in Milan.
Martin Andrews, Head of the Rare Disease Unit, GSK said: “Today’s approval is the result of many years’ work with our collaborators in Milan and is the next step towards bringing life-changing treatment to patients with ADA-SCID and their families. This is the start of a new chapter in the treatment of rare genetic diseases and we hope that this therapeutic approach could also be used to help patients with other rare diseases in the future.”
The marketing authorization decision was based on data collected from 18 children treated with Strimvelis. A 100% survival rate at three years post-treatment with Strimvelis was observed for all children in the pivotal study (n=12) and every child receiving the treatment who contributed to the marketing authorization data package is alive today (n=18), with a median follow-up duration of approximately seven years. Full results of the analysis have recently been published in BLOOD1.
Professor Alessandro Aiuti, Clinical Research Coordinator at San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), a joint research collaboration between OSR and Telethon, said: “We are delighted with today’s news, which marks the culmination of more than 20 years of research and development at Tiget. This innovative, individualized treatment approach uses a patient’s own gene modified stem cells to correct the root cause of the disease. It has been gratifying for all of us to see patients affected by this severe immune deficiency growing over the years, being able to play with other children and going to school. Working alongside GSK, we can now make Strimvelis available to ADA-SCID patients and transform the lives of children who so desperately need it.”
Nicola Bedin, CEO of Ospedale San Raffaele, said: “This great achievement would not have been possible without the effective collaboration between OSR, Telethon and GSK, which has brought together years of scientific research, first-class medical practice and expertise in product development. Going forward we hope to build on our shared mission to develop and deliver more much-needed new medicines to patients with rare diseases.”
Francesca Pasinelli, General Manager of Fondazione Telethon, said: “This is a memorable day, not only for us, but overall for the people we work for: with Strimvelis we can keep our promise to patients. We can say that we have pioneered a model whereby the charity organisation acts not only as a funding agency, but plays a primary role in managing the development of research to ensure that each step of the process leads to the ultimate goal, which is to provide accessible therapy to patients.”
Source: gsk
