In late October 2021, the National Institute for Care and Excellence (NICE) in England recommended Givlaari (givosiran) to treat patients with acute porphyria — or, more specifically, to treat patients undergoing porphyria attacks. Now, just shortly after, the country’s National Health Service (NHS) has shared that it will be offering access to the treatment for patients. According to Porphyria News, NHS Givlaari access should begin by February 2022.
Givlaari
So what exactly is Givlaari? According to Alnylam Pharmaceuticals (“Alnylam”), Givlaari is:
the world’s first-ever approved GalNac-conjugate RNAi therapeutic [that] was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits, or intravenous (IV) hemin administration at home. Givlaari works by specifically reducing the elevated levels of aminolevulinic acid synthase 1 (ALAS1) messenger RNA (mRNA) in the liver, leading to reduction of neurotoxins associated with attacks.
The therapy is given once monthly and administered subcutaneously. NHS access will be available for patients who experience at least four porphyria attacks (or more) within a 1-year span. Additionally, the medication will only be provided at specific expert centers.
Over the coming months, the NHS will be working to ensure that patients will receive access to Givlaari treatment.
Porphyria
There are two forms of porphyria, disorders characterized by chemical accumulation which overproduces porphyrin: acute and cutaneous. While acute porphyria impacts the nervous system, cutaneous impacts the skin. Normally, porphyrin plays a role in hemoglobin function. However, when too much porphyrin is produced, it manifests in health issues. Additionally, both forms of porphyria are characterized by an enzyme deficiency preventing the body from producing heme. Risk factors for porphyria or porphyria attacks include sunlight exposure, being female, smoking or using alcohol, menstrual hormones, or stress. Symptoms vary based on the form of porphyria. You can learn more about the symptoms of cutaneous porphyria by clicking the link below, but since Givlaari is designed for the acute form, symptoms of acute porphyria include:
- Diarrhea or constipation
- Nausea and vomiting
- Abdominal, chest, leg, and back pain
- Muscle weakness, numbness, pain, or paralysis
- Fatigue
- Rapid heartbeat and/or heart palpitations
- High blood pressure
- Anxiety/depression
- Restlessness and/or difficulty sleeping
- Red or brown urine
- Mental changes, such as confusion, paranoia, or hallucinations
- Seizures
- Chronic pain (long-term complication)
- Liver damage (long-term complication)
- Chronic kidney failure (long-term complication)
Learn more about porphyria.
