Making the RARE voice heard
New real world evidence shows that an extended half-life product can reduce the number of bleeding episodes and improve quality of life for those living with haemophilia
New evidence from real-life clinical practice in the UK, presented today at the World Federation of Haemophilia (WFH) 2018 World Congress in Glasgow, shows that people with haemophilia A treated with an extended half-life (EHL) clotting factor have fewer bleeds than with previously available treatments.[i]
The new findings, based on an analysis of the UK National Haemophilia Database, indicate that treatment with efmoroctocog alfa factor VIII (rFVIIIFc, Elocta®), led to a reduction in bleeding episodes in patients living with haemophilia A.i The median annualised bleed rate (ABR) among 70 haemophilia A patients fell from 1.5 (interquartile range (IQR) 0.0-6.0) a year when treated with conventional clotting factor VIII for a median of 58 weeks to 1.3 ( IQR 0.0-3.5) a year when treated with rFVIIIFc (p=0.6) for a median of 41 weeks.i However, among 21 patients who had an ABR ≥5, the median ABR fell from 10.4 (IQR 7.2-12.5) to 3.3 (IQR 2.2-4.4)(p<0.01).i
In addition, the number of infusions among the 70 haemophilia A patients fell from a median of 3.5/week when treated with conventional clotting factor VIII to 2.4/week when treated with rFVIIIFc, a 33% reduction (p<0.0001), representing an average of 57 fewer infusions per year.i Infusion time varies depending on the weight of the patient and the dose they need. However, the whole infusion process can take several hours per week for families with more than one child with haemophilia. Therefore a reduction in the number of infusions can be of great benefit to those living with haemophilia and their carers, reducing the burden of treatment and making it easier to go about day-to-day life without interruption.
Furthermore, a reduction of factor usage of 14% was seen among all haemophilia A patients in the study treated with rFVIIIFc, from a median of 95 IU/kg/week to 82 IU/kg/week (p<0.0001).i This change was all the greater among those patients with an ABR ≥5, for whom factor usage decreased by 23% (p<0.01).i
It has been found that most bleeding in haemophilia occurs internally, into the joints or muscles,[ii] which can cause pain and discomfort; even one joint bleed can cause permanent damage.[iii] Liz Carroll, CEO, The Haemophilia Society, commented:
“A survey of our members has told us that a reduction in joint problems, caused by bleeds, would be the most important factor for improving their quality of life, so it is reassuring to see that EHL treatments are having this effect. In families in which several members are living with haemophilia, the time saved from having fewer infusions also impacts on family life.”
We’re delighted to see that the real world evidence matches what has already been seen in clinical trials, that treatment with our EHL product reduces the number of bleeding episodes for someone living with haemophilia A
Neil Dugdale, Vice-President and General Manager, Sobi UK and RoI
Neil Dugdale, Vice-President and General Manager, Sobi UK and RoI, commented: “We’re delighted to see that the real world evidence matches what has already been seen in clinical trials, that treatment with our EHL product reduces the number of bleeding episodes for someone living with haemophilia A.”
Haemophilia is a rare, genetic disorder in which the ability of a person’s blood to clot is impaired. About 6,000 people are affected with haemophilia in the UK; approximately 5,400 people have haemophilia A and approximately 1,100 people have haemophilia B.[iv] Sobi’s rFVIIIFc is a recombinant clotting factor therapy, which replaces the missing clotting factor in the body. It uses Fc fusion technology to make it possible to achieve much higher clotting factor levels in the body than with the same dose of traditional, short-acting, treatments. The product also uses naturally occurring pathways to extend the time the therapy remains in the body, hence the name ‘extended half-life’ product.
Sobi™ is an international speciality healthcare company dedicated to rare diseases. Our vision is to be recognised as a global leader in providing access to innovative treatments that make a significant difference for individuals with rare diseases.
The product portfolio is primarily focused on treatments in Haemophilia and Specialty Care. Partnering in the development and commercialisation of products in specialty care is a key element of our strategy. Sobi has pioneered in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2017, Sobi had total revenues of SEK 6.5 billion and approximately 850 employees. The share (STO:SOBI) is listed on Nasdaq Stockholm. More information is available at www.sobi.com.
About haemophilia A and B
Haemophilia is a rare, genetic disorder in which the ability of a person's blood to clot is impaired. Haemophilia A occurs in about one in 5,000 male births annually, and more rarely in females. Haemophilia B occurs in about one in 25,000 male births annually, and more
rarely in females. The World Federation of Hemophilia estimates that approximately 180,000 people are currently diagnosed with haemophilia A and B world-wide[v]
People with haemophilia A and B experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages. Prophylactic injections of factor VIII and factor IX can temporarily replace the clotting factors that are needed to control bleeding and prevent new bleeding episodes. The World Federation of Hemophilia (WFH) recommends prophylaxis as the optimal therapy as it can prevent bleedings and joint destruction.
[i] Martin Scott, Hua Xiang, Peter W Collins, Charles R M Hay. The effect of switching to rFVIIIFc on treatment patterns and annualised bleed rate before and after: a within-patient comparison from the UK National Haemophilia Database, abstract no W-FPYR-005 (135), presented at WFH World Congress 23 May 2018, Free Papers: Young Researchers Session, 16.30-18.00, Glasgow, UK.
[ii] World Hemophilia Society. About bleeding disorders: Frequently asked questions. http://www.wfh.org/en/page.aspx?pid=637
Accessed April 2018
[iii] Gringeri A, Ewenstein B, Reininger A. The burden of bleeding in haemophilia: is one bleed too many? Haemophilia. 2014;20:459–463.
Rare Revolution Editor