Borders drug outfit seeks approval for rickets treatment
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The Galashiels firm, owned by Japan’s Kyowa Hakko Kirin, said the move marks a “major milestone” for the company as the new drug, dubbed KRN23, is the first in its pipeline of treatments to reach the stage of seeking European approval.
Rickets, which can cause bone pain, poor growth and deformities such as bowed legs and curvature of the spine, is most commonly linked to a lack of calcium or vitamin D.
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Hide AdHowever, it can also be triggered by X-linked hypophosphatemia (XLH), a rare genetic disorder that affects about one in 20,000 people, whose kidneys treat phosphorus as a waste product instead of returning enough of it to the circulation for use by bones and teeth.
KKI chief executive Tom Stratford said: “XLH is a debilitating condition which causes long-term pain and distress among sufferers and for which there are no currently approved treatments that target the underlying cause.
“As part of Kyowa Hakko Kirin, we strive to contribute to the health and wellbeing of people around the world through advances in life sciences and technologies. The acceptance of this filing brings us one step closer to addressing the unmet medical needs of patients who suffer from XLH.”
KKI is collaborating with Ultragenyx Pharmaceutical, a US-based firm that targets rare genetic diseases, in the development and commercialisation of KRN23. A decision from the European Medicines Agency’s committee for medicinal products for human use is expected in the second half of the year.
Ultragenyx chief executive Emil Kakkis said: “Based on the positive results from multiple studies in paediatric and adult patients with XLH, we are pursuing conditional marketing authorisation in order to accelerate access to this therapy for patients.”