Early Access Programme for KKI

Spreng Thomson blog

FIRST EARLY ACCESS PROGRAMME INITIATED FOR KYOWA KIRIN INTERNATIONAL’S INVESTIGATIONAL TREATMENT FOR X-LINKED HYPOPHOSPHATEMIA (XLH)

  •  Kyowa Kirin International’s burosumab (KRN23) is an investigational recombinant fully human monoclonal IgG1 being evaluated by the European Medicines Agency as the first potential treatment for XLH
  • Germany’s Paul-Ehrlich-Institute, the Federal Institute for Vaccines and Biomedicines has confirmed the notification for compassionate use of burosumab

 

Galashiels, Scotland — 24 July, 2017 — Kyowa Kirin International PLC, a wholly owned subsidiary of Kyowa Hakko Kirin Co., Ltd. (Kyowa Hakko Kirin) today announces Germany’s Federal Institute for Vaccines and Biomedicines has confirmed the notification through the Ordinance on Medicinal Products for Compassionate Use (Arzneimittel-Härtefall-Verordnung, AMHV) to make burosumab available to eligible paediatric patients with X-Linked Hypophosphatemia* (XLH).  Burosumab is an investigational treatment for XLH.[i] This notification means that eligible paediatric XLH patients in Germany can now have access to burosumab. This is the first Early Access Programme to be launched for XLH worldwide.

The Ordinance on Medicinal Products for Compassionate Use aims to give people with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation, and has only been granted to a limited number of products since its inception in 2014.[ii]

XLH is a rare, genetic bone disorder characterised by urinary phosphate wasting due to excess production of FGF23.[iii] Children with XLH may have rickets, progressive bowing of the leg, osteomalacia, bone pain, waddling gait, short stature, gross motor impairment, muscle weakness, frequent/poorly healing pseudofractures, spinal stenosis, enthesopathy and osteoarthritis.[iv],[v],[vi]  It is estimated that prevalence of XLH in children is approximately one in every 20,000.[vii]

“This notification is an important step for burosumab and we are proud to have entered into the voluntary Ordinanace on Medicinal Products for Compassionate Use” said Dr. Tom Stratford, President and CEO of Kyowa Kirin International.

*XLH is also known as “Phosphatdiabetes” in Germany.

 

[i] A Study of KRN23 for X-linked hypophosphatemia. Available at: https://clinicaltrials.gov/ct2/show/NCT00830674?cond=XLH&draw=1&rank=7 Last accessed July 2017.

[ii] Paul-Ehrlich-Institut. Available here: http://www.pei.de/EN/information/license-applicants/clinical-trial-authorisation/compassionate-use/compassionate-use- Last accessed July 2017.

3 Carpenter TO et al. A clinician’s guide to X-linked hypophosphatemia. J Bone Miner Res. 2011;26:1381–1388. Available here: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3157040/ Last accessed July 2017.

[iv] Carpenter TO, Imel E, Ruppe M et al. Randomized trial of the anti-FGF23 antibody KRN23 in X-linked hypophosphatemia. Journal of Clinical Investigation 2014;124(4):1587-1597.  Available here: https://www.ncbi.nlm.nih.gov/pubmed/24569459 Last accessed July 2017.

[v] Ming Yang H, Mao M, Yang F et al. Recombinant growth hormone therapy for X-linked Hypophosphatasemia in children. Cochran Cystic Fibrosis and Genetic Disorders Group 2005; doi:10.1002/14651858.CD004447.pub2.

[vi]  Beck-Nielsen et al. Incidence and prevalence of nutritional and hereditary rickets in southern Denmark Eur J Endocrinol 2009;160:491.

[vii] Beck-Nielsen et al. Incidence and prevalence of nutritional and hereditary rickets in southern Denmark Eur J Endocrinol 2009;160:491.