Givlaari, a treatment for acute hepatic porphyria (AHP), was first approved in Europe back in March of 2020. However, approval doesn’t mean immediate access unfortunately. It’s been nearly two years since this approval, and Givlaari is just now availability imminent.
The National Institute for Health and Care Excellence (NICE) controls pricing for drugs in England, and they previously rejected Gilvaari’s price of $575,000 each year ($39,000 for a singular vial). Now, they’ve reversed this decision. Alnylam, the creator of the treatment, has agreed to provide the treatment at a discount (albeit confidential), and it will become available through the National Health Service (NHS).
AHP
AHP is a rare genetic condition which impacts the liver. It causes a buildup of porphyrins within the liver, which can lead to seizures, nausea, and vomiting. Unfortunately, AHP can also lead to other conditions including insomnia, issues with the heart and lungs, and even paralysis.
In Europe, 1 in every 100,000 individuals are affected and there are around 560 with the diagnosis in England alone. Although the condition is not constant, recurrent attacks take a toll on 10% of patients.
In the UK, there are approximately 17 new patients diagnosed each year, and 27 people each year face recurrent attacks. This condition can severely impact quality of life, cause frequent hospitalizations, and immense, daily pain.
Givlaari
Gilvaari is a treatment for those facing the most severe presentation of AHP. These individuals experience four or more seizures each year. Although there is another available treatment for these individuals with severe disease called Normosang, the therapy is no longer effective after patients use it for a few years.
This new treatment uses gene silencing, which means it targets the production of the porphyrins, attacking the root cause of the disease. Researchers are extremely hopeful about what this treatment could mean to patients and how it could improve their quality of life.
You can read more about this new treatment and its upcoming NHS availability here.
