Swedish_Orphan_Biovitrum_Sobi

First treatment for babies with ultra-rare spectrum of autoinflammatory diseases now available in UK

In Press Release by CameronLeave a Comment

Swedish_Orphan_Biovitrum_Sobi

  • Anakinra (Kineret®) now offers treatment option for children from eight months old with cryopyrin-associated periodic syndromes (CAPS)

  • Now supplied in a syringe with marked graduations to allow for dose adjustment in paediatric patients

April 23, 2014

London: Anakinra (Kineret®), an interleukin-1 (IL-1) inhibitor, is now available in the UK for treatment of CAPS in children and adults. Anakinra is the first CAPS treatment licensed for use in infants as young as eight months by the Medicines and Healthcare Products Regulatory Agency (MHRA).

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. Patients suffer substantial pain and progressive reduction in their quality of life. The most severe form of the condition causes chronic aseptic meningitis. Late complications include hydrocephalus, developmental delay, mental retardation, and hearing loss.1

Despite severe symptoms, children with CAPS typically experience a long journey to diagnosis. This plight is common for people with rare diseases, and it is hoped that the national rare disease strategy being implemented by the four home governments will help reduce delays to diagnosis and ensure that more patients are offered coordinated care close to home. Although numbers of patients with individual rare diseases are small, when grouped together their sufferers are a significant and under-served population; 1 in 17 people in the UK will be diagnosed with rare disease.2

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 wait years for a diagnosis, and as a result of the rarity of their disease, may have to work hard to get appropriate treatment. Clinicians in secondary care should suspect an autoinflammatory condition such as CAPS – and consider referring to a tertiary rheumatology centre – if they encounter unexplained fevers, rash, headaches and joint pain, especially if symptoms do not respond to standard care.”

Until now, the only licensed treatment was for patients over 2 years old, and patients have to travel to London to receive this treatment. Patients can now choose to have their therapy managed at home with daily injections, monitored by their local specialist.

Nicky Freeman, Paediatric Rheumatology Nurse Specialist at Birmingham Children’s Hospital commented: “Working closely with CAPS patients and their families you see that travelling long distances for treatment isn’t always feasible, which is why it’s great to have the option to manage their treatment at home, with shared care at a local hospital coordinated by the tertiary hospital.”

To facilitate use in paediatric patients, anakinra is now supplied in a syringe with marked graduations to allow for dose adjustments. Anakinra has a well-documented safety profile. It inhibits IL-1, an inflammatory cytokine which is implicated in a number of rare inflammatory conditions.

CAPS affects approximately 1 in 1,000,000 people in the UK.3

 

References:

  1. Hashkes, PJ (2012) Autoinflammatory disease. Pediatric Clinics of North America
    Volume 59, Issue 2 , Pages 447-470.
  2. http://www.raredisease.org.uk/about-rare-diseases.htm Accessed 04.04.14
  3. National Specialised Commissioning Team. National treatment service for cryopyrin associated periodic fever syndromes (CAPS). Service Specification 2012/13.

Notes to editors:

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 1:1,000,000.3

 

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® is supplied ready for use in a graduated pre-filled syringe that 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.

 

About Sobi

Sobi is an international specialty healthcare company dedicated to rare diseases. Our mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primarily focused on inflammation and genetic diseases, with three late stage biological development projects within haemophilia and neonatology. We also market a portfolio of specialty and rare disease products for partner companies. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2012, Sobi had total revenues of SEK 1.9 billion (€ 215 M) and about 500 employees. The share (STO: SOBI) is listed on NASDAQ OMX Stockholm. More information is available at www.sobi.com.

 

About the National Strategy for Rare Diseases

The ‘UK Strategy for Rare Diseases’ contains over 50 commitments to ensure people living with a rare disease have access to the best evidence-based care and treatment that health and social services, working with charities, researchers and industry, can provide.

The UK commitments are in 5 areas:

-          Making sure patients and their families and carers have the information they need, are listened to and consulted

-          Developing better methods of identifying and preventing rare diseases

-          Improving diagnosis and earlier intervention for those with a rare disease

-          Developing better coordination of care for those with a rare disease, including joined up consultation and treatment schedules

Building on research to improve personalised approaches to healthcare for those with a rare disease

Leave a Comment

Current ye@r *