In the US, the first treatment of the most common form of dwarfism was approved. However, Britain will need to wait until 2023 for it to be made available to them on their NHS.
Vosoritide, a once-daily injection, was given the green light by the US Food and Drug Administration (FDA) for use on children over five-years-old with achondroplasia.
The drug approval was approved by the US Health Authorities and will soon be available for purchase starting in mid-December.
Achondroplasia can be the cause of eight in ten dwarfism cases. It affects one in 25,000 babies each year and can result in life-threatening conditions, such as fluid buildup in the brain. But, most people will have normal life expectancies.
Children will be given the injection — which costs around £240,000 ($320,000) per patient — over the course of a year.
It reverses the genetic flaw that makes a gene involved in bone growth overactive. The drug stops this from happening.
The results of a clinical trial with vosoritide were published last September. They showed that patients receiving the drug grew on average one-half inch faster than children who received a placebo.
Part of the global trial was conducted in Guy’s and St Thomas NHS Trust in London.
Vosoritide continues to undergo a cost-effectiveness and clinical evaluation by NHS’s medical watchdog. An update on the National Institute for Health and Care Excellence (NICE) website said the next stage of the process won’t go ahead until 2023.
MailOnline reached out to NICE for further information.
The US Food and Drug Administration has approved a drug that treats the most common form of dwarfism but patients with the condition in the UK may have to wait until 2023 before it could be approved for use on the NHS
There are about 7,000 people with a form dwarfism in the UK, the majority of which is achondroplasia, thought is unknown how many might be eligible for the vosoritide treatment if it was approved in the UK.
One in every 25,000 babies is affected by Achondroplasia, which means that approximately 28 children in the UK are born with this condition every year.
NICE updated in November 2013: “Following an update by the company that develops vosoritide,” NICE stated that it had decided that a scoping exercise was not necessary at this point.
“The scoping exercise would be rescheduled and will take place later in 2023.
NICE did not provide any more details on the nature of the update from the company that makes vosoritide, by American biotechnology BioMarin Pharmaceutical, which had prompted the delay.
Youroritide appraisal in the UK is delayed This makes it an exception to the rest of its neighbours. In August, the EU approved this drug for use.
Theresa Kehoe (FDA’s Director of General Endocrinology) said that the approval will benefit more than 10,000 US children.
“With this action, children suffering from achondroplasia with short stature have an option to treat the root cause.
BioMarin Pharmaceutical chief executive Jean-Jacques Bienaimé welcomed the FDA’s approval and said the drug will be available in the US by the middle of next month.
He said, “We are grateful to the FDA for recognizing its potential as a therapeutic option for children suffering from achondroplasia,”
The company also stated that it seeks approval in Japan, Australia, and Brazil for the drug. However they did not mention any plans to do so in the UK.
Vosoritide has been controversial in the dwarfism community, with some organisations arguing the focus on treating height and not other health conditions that can occur with achondroplasia is unhelpful.
Health problems associated with achondroplasia such hearing loss, sleep apnea, and life-threatening skeletal and problems with fluid in the brain.
Restricted Growth Association UK previously stated that it wouldn’t support the drug if participants were only able to attain an extra level of height.
Achondroplasia occurs in approximately one in every 255,000 births worldwide. The condition causes a person to have normal sized trunk or torso, but smaller limbs.
A large, prominent head, a flattened nose bridge, and a curvier lower spine can be common. They also may have exceptionally flexible joints.
Achondroplasia affects adults at an average height of four feet. The majority are children with average-sized parents.
While children with achondroplasia may develop some motor skills and mobility more slowly because of the combination of a heavier head and shorter arms and legs, both life expectancy and intelligence are not affected.
This is the leading cause of dwarfism, accounting for seven to eight of ten cases.