NICE (The National Institute of Clinical Excellence), the drug decision making body in England, have publicly released an update today (2 February 2022) about elosulfase alfa (Vimizim®) for the treatment of individuals with MPS IVA. The update can be found below.
"Following on from the committee meeting on the 13 January 2022, we can confirm that the company (BioMarin) and NHS England have commenced commercial discussions. A further update will be provided by NICE before the end of February 2022."
The MPS Society will update our MPS IVA Morquio community as soon as we hear anything further.
About MPS IVA and treatment
MPS IVA is a rare and progressive disease, that causes a wide spectrum of symptoms that severely impact the quality of life of people with the condition, and their families and carers. Patients have short stature with joint and skeletal abnormalities, respiratory symptoms including airway obstruction, hearing loss, corneal clouding and heart valve abnormalities. The condition also causes pain, fatigue, progressive loss of endurance and increasing dependence on a wheelchair. It leads to reduced life expectancy, primarily through respiratory failure and heart problems.
Before elosulfase alfa, the only treatment option was standard care, which aims to relieve symptoms and included several treatments such as corticosteroids and bronchodilators to improve lung function and spinal and orthopaedic surgeries. Since December 2015, the majority of eligible patients have joined the managed access agreement and received treatment with elosulfase alfa and met the criteria to remain on treatment. These criteria set out both clinical and quality of life measures designed to ensure that treatment was directed to those who derived benefit from it.
About the MPS Society
If you would like to talk to a member of our Support and Advocacy team, please call 0345 389 9901 or email firstname.lastname@example.org
If you'd like to read more about NICE's evaluation process, please click here.