GSK files ultra-rare disease application with EMA
pharmafile | May 6, 2015 | News story | Research and Development, Sales and Marketing |Â Â EMA, GSK, GSK2696273, Wiskott-Aldrich syndrome, bubble boy, metachromatic leukodystrophyÂ
GlaxoSmithKline has become the first big pharma firm to file for a European marketing authorisation for a gene therapy to treat a rare immune disease affecting children.
The British firm, along with Italian research partners Fondazione Telethon and the San Raffaele Hospital in Milan, filed an application with the European Medicines Agency for GSK2696273.
The investigational gene therapy is a potential treatment for adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID) – also known as ‘bubble boy disease’ – for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
ADA-SCID is an ultra-rare disease affecting only a dozen or so children in Europe every year. Those born with this condition have a faulty gene inherited from both parents that stops them producing an essential protein called adenosine deaminase (ADA), which the body needs to make lymphocytes (a type of white blood cell).
As a result, children with ADA-SCID have a severely deficient immune system, and common infections can result in severe and life-threatening illness. Many children do not survive beyond two years old.
Normally treatment involves using anti-infectives to prevent infections, and in the longer-term a bone marrow transplant if this is available from a matched donor. Gene therapy – if approved – will provide another option.
Treatment with GSK2696273 involves collecting stem cells from the patient, and in the lab, using a type of virus called a retrovirus which is inserted into the stem cells to correct the faulty gene. The modified cells are then fed back into the patient through a drip, and low-dose chemotherapy can increase the chances of successful treatment.
GSK2696273 is borne out of GSK’s strategic alliance with San Raffaele Hospital and Fondazione Telethon. It also gives GSK the exclusive option to develop six products using the gene transfer technology developed by scientists at the Raffaele Telethon Institute for Gene Therapy.
Researchers from the same team conducted the research on which the EMA application is based. They have investigated the treatment in 18 children treated in two pilot studies, a Phase I/II clinical study and a named patient, early access programme. All are alive today and there have been no significant events reported to date.
Patrick Vallance, president of pharmaceuticals R&D at GSK, said: “I am very pleased that we are now at a stage to file for approval of this gene therapy for ADA-SCID – a devastating rare disease which drastically limits and shortens patients’ lives.
“We believe this marks a significant milestone, showing the potential of gene therapy as an important additional modality for tackling the underlying cause of serious diseases.”
GSK has already exercised its option to develop two further programmes in rare diseases called metachromatic leukodystrophy (MLD) and Wiskott-Aldrich syndrome (WAS), which are both currently in clinical trials.
Lilian Anekwe
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