followers 4 popularity
following 0

followers  view all

elizabethjennifer is not in any groups

First treatment for infants with CAPS licensed in UK

Jan 1st 2014 at 8:58 PM

Anakinra (Kineret®), an interleukin-1 (IL-1) inhibitor, has been granted a licence for treatment of CAPS in children and adults by the Medicines and Healthcare Products Regulatory Agency (MHRA). Anakinra is the first CAPS treatment licensed for use in children as young as eight months. CAPS is an umbrella term for a number of ultra-rare, life-long, severely debilitating autoinflammatory diseases which cause rash, headache, fever, joint pain and other inflammatory symptoms.

CAPS affects approximately 1 in 1,000,000 people in the UK.1 Patients suffer substantial pain and progressive reduction in their quality of life. The most severe form of the condition causes chronic inflammation of the meninges (aseptic meningitis). Late complications include hydrocephalus, developmental delay, mental retardation, and hearing loss.2 Dr Paul Brogan, Reader in Vasculitis and Honorary Consultant Paediatric Rheumatologist at the UCL Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, commented:

"Paediatricians looking after young children with CAPS are delighted that medicines to treat this condition are increasingly available to be used in patients from very early in life. We believe that early diagnosis, and institution of appropriate treatment in a timely fashion will reduce suffering, and long-term complications of this genetic disease." Patients now have the choice of two licensed treatments for CAPS via the National Amyloidosis Centre in London - the national centre of excellence for this group of conditions.

Patients choosing anakinra will be able to have their therapy managed at home with daily injections, monitored by their local specialist. Dr Liza McCann, Consultant Paediatric Rheumatologist at Alder Hey Children's Hospital said: "It isn't always practical for CAPS patients to regularly travel extensively for treatment, so it is really valuable for patients and clinicians to have choice of treatment, including the option of managing the condition at home with anakinra." Anakinra inhibits IL-1, an inflammatory cytokine, and is used to treat a number of rare inflammatory conditions. It has a well-documented safety profile. IL-1 inhibitors have shown similar efficacy in several autoinflammatory conditions, although no head-to-head studies have been conducted to date.

Professor Taunton Southwood, Professor of Paediatric Rheumatology, University of Birmingham and Paediatric Rheumatologist at Birmingham Children's Hospital welcomed the approval of anakinra, saying: "While the number of patients with CAPS is small, it has a heavy burden on the lives of those affected. These are patients who often struggle to get a diagnosis, and as a result of the rarity of their disease, may have to work hard to get appropriate treatment. Any development which helps more of these patients access treatment that better fits into their lifestyle is very positive." To facilitate use in paediatric patients, the new licence requires use of a syringe with marked graduations to allow for dose adjustments. It is anticipated that this graduated syringe will be available, allowing clinicians to use anakinra in CAPS, in spring 2014. About CAPS Cryopyrin-associated periodic syndromes (CAPS) are a group of rare inherited autoinflammatory diseases caused by autosomal dominant mutations in a gene called NLRP3. CAPS is characterized by uncontrolled overproduction of IL-1 beta. IL-1 induces a number of inflammatory responses such as fever, pain sensitisation, bone and cartilage destruction and acute plasma protein responses. In the most severe form of CAPS, called neonatal-onset multisystem inflammatory disease (NOMID, also known as chronic infantile neurologic cutaneous and arthritis syndrome, CINCA), it is associated with increased mortality and fever, rash, chronic aseptic meningitis, sensorineural hearing loss, craniofacial abnormalities, and bone lesions. In the intermediate severity form, called Muckle-Wells syndrome (MWS), the disease is typically associated with episodic, intense and enduring flares, and increasing morbidity, including progressive hearing loss and kidney failure secondary to amyloidosis (a condition where amyloid proteins are deposited in organs and/or tissues). The mildest form, called familial cold autoinflammatory syndrome (FCAS), presents with cold-induced episodes of fever, rash and malaise. The incidence of CAPS is estimated to be 1:1,000,000.1 About anakinra (Kineret®) Kineret® is a recombinant protein drug that blocks the biological activity of IL-1 by binding to the IL-1 type 1 receptor, expressed in a wide variety of tissues and organs. IL-1 is a key mediator of inflammation and driver of autoinflammatory diseases in both adults and children. Kineret® is indicated in adults, adolescents, children and infants aged eight months and older with a body weight of 10 kg or above for the treatment of CAPS, including: Neonatal-onset multisystem inflammatory disease (NOMID) / chronic infantile neurological, cutaneous, articular syndrome (CINCA) Muckle-Wells syndrome (MWS) Familial cold autoinflammatory syndrome (FCAS) The recommended starting dose in all CAPS subtypes is 1-2 mg/kg/day by subcutaneous injection. Kineret® will be supplied ready for use in a graduated pre-filled syringe (anticipated availability March 2014). The graduated pre-filled syringe allows for doses between 20 and 100 mg. Kineret® is also indicated in adults for the treatment of the signs and symptoms of Rheumatoid Arthritis (RA) in combination with methotrexate, with an inadequate response to methotrexate alone. For more information on Kineret® see the Summary of Product Characteristics.

Please to comment

sign in

Remember Me

New to IM faceplate? join free!

Lost Password? click here