A Borders pharmaceutical plant believes it could be about to make a breakthrough in treating rickets as it seeks authorisation for a new drug for sufferers of the most common heritable form of the condition.
Galashiels-based Kyowa Kirin International (KKI) has developed an antibody for the genetic condition X-linked hypophosphatemia.
The condition it hopes to treat, a deforming and painful form of rickets affecting the bones and growth of children, does not respond to vitamin D treatment, so new medication is required.
The drug, KRN23, is the first in a series produced by KKI’s parent company, Kyowa Hakko Kirin, to reach the stage of seeking European approval.
The firm says the European Medicines Agency has accepted its marketing authorisation application for the drug for review, and an opinion from the committee for medicinal products for human use is expected in the second half of this year.
The company says the new drug is part of its efforts to “contribute to the health and wellbeing of people around the world”.
Tom Stratford, president and chief executive officer of KKI, said: “X-linked hypophosphatemia 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.”
He added: “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 X-linked hypophosphatemia.”
Rickets is caused by vitamin D deficiency, resulting in weakened bones.
Though the strain of the condition the drug is aimed at tackling is the most common heritable form of rickets, it is still rare in that it currently only affects around 16,000 people across Europe.
The strain XLH is a disorder of phosphate metabolism caused by phosphate-wasting in urine, leading to severe hypophosphatemia, which can be inherited as an X-linked dominant trait affecting both males and females. Currently, most sufferers are managed using oral phosphate replacement and active vitamin D therapy, requiring multiple doses daily and continual monitoring of potentially serious side-effects.
The new drug being developed in Galashiels, however, could change the way the illness is treated in the near future and cut down on the amount of time spent treating it.
Formerly known as ProStrakan, a firm founded by Dr Stratford’s father Harry in Melrose nearly 22 years ago, KKI now employs almost 150 staff at its Galashiels base.
ProStrakan was taken over by the Japanese firm KHK in 2011 and rebranded as part of a move to become a global speciality pharmaceutical company.
KHK’s western pharmaceutical subsidiaries, including specialist divisions employing another 350 people outwith the Borders, also came under the Kyowa Kirin name, and since the takeover 20 new staff, including several qualified medics, have joined its Galashiels workforce.
The company, a subsidiary of Kyowa Hakko Kirin, has previously developed a number of new products which have been licensed and trialed across Europe and in the United States of America.
KKI is headquartered in Scotland.