According to FirstWord Pharma, patients with paroxysmal nocturnal hemoglobinuria (PNH) in the United Kingdom (UK) may soon have a more effective and efficient, and less invasive, treatment option. Currently, the standard-of-care for PNH requires eculizumab injections every 2 weeks. However, using ULTOMIRIS (ravulizumab), these infusions are only needed every 8 weeks. The National Institute for Health and Care Excellence (NICE) has now recommended that the National Health Service (NHS) adopt ULTOMIRIS for patients with PNH.
Developed by biopharmaceutical company Alexion Pharmaceuticals (“Alexion”), ULTOMIRIS is described as:
the first and only long-acting C5 inhibitor administered every 8 weeks in adults. In maintenance dosing, ULTOMIRIS works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system.
Normally, the complement cascade plays a role in immune response. But if that response is over-active, it can cause the immune system to attack the body, causing health issues. Outside of PNH, ULTOMIRIS is also approved for both pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS).
The recommendation from NICE comes following a review of both health economic and clinical data, insights from experts, and information offered by a variety of advocacy organizations. Ultimately, patients deserve an opportunity to improve their quality of life (QOL). With less frequent, but similarly effective treatment options, ULTOMIRIS might offer an avenue to achieve this.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and sporadically acquired hematopoietic stem cell disorder which causes early and abnormal red blood cell destruction. Overall, PIGA gene mutations cause PNH. Through these mutations, the body develops rapidly multiplying PNH cells, which crowd out healthy platelets, as well as red and white blood cells. As a result, PNH is characterized not only by red blood cell destruction but by bone marrow abnormalities and blood clots. Typically, PNH affects younger individuals. While hemolysis (the destruction of red blood cells) is continual, it may worsen during times of stress. On average, symptom onset occurs between ages 35-40. Symptoms of PNH include:
- Dark or bloody urine
- High heart rate
- Anemia (low red blood cell count)
- Easy bruising and bleeding
- Chest pain
- Shortness of breath and/or difficulty breathing
- Abdominal pain and cramping
- Male sexual dysfunction
- Difficulty swallowing
- Kidney disease