GlaxoSmithKline’s Strimvelis recommended for ACA-SCID in NICE draft guidance

GlaxoSmithKline's Strimvelis has been recommended as a treatment for an ultra-rare inherited immune deficiency in provisional guidance from the National Institute for Health and Care Excellence (NICE).

The UK regulator has issued final draft guidance recommending Strimvelis for availability on the NHS as a therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID), a condition that often results in children having to live in isolation.

Colloquially known as "bubble baby syndrome", the disease affects around three babies in England each year and results in a severely compromised immune system, meaning children need to be kept in a controlled environment at all times.

Strimvelis is the first therapy for a very rare disease to be recommended under NICE's new, higher cost-effectiveness limits, with the regulator backing the use of the treatment when no suitable matched related stem cell donor is available.

The treatment is usually given only once and delivers lifelong effects. It has to be given at a specific hospital in Milan, meaning patients will travel to Italy to receive the treatment.

Professor Carole Longson, director of the centre for health technology assessment at NICE, said: "Strimvelis represents an important development in the treatment of ADA-SCID, offering the potential to cure the immune aspects of the condition and avoid some of the disadvantages of current treatments."

With over 20 years of experience within the pharmaceutical market, we at Zenopa have the knowledge, skills and expertise to help find the right job for you. To find out more about the current pharmaceutical roles we have available, you can search for the latest job roles, register your details, or contact the team today.