Velaglucerase alpha for injection, commonly known as VPRIV, is a long-term enzyme replacement therapy (ERT) for children and adults with type 1 Gaucher disease. VPRIV was developed by Shire Human Genetic Therapies (part of Takeda Pharmaceutical Company) and produced using a proprietary method of gene activation.
How VPRIV works
In type 1 Gaucher disease, deficiency of an enzyme called glucocerebrosidase (GCase), due to mutations in the GBA1 gene, results in the accumulation of a fatty chemical called glucocerebroside in the bone marrow, liver, and spleen. Type 1 Gaucher patients have low red blood cell and platelet counts, making them susceptible to bleeding and bruising as well as enlarged livers and spleens.
VPRIV contains the same enzyme as naturally occurring GCase, which is deficient in type 1 Gaucher disease patients. Thus, it can supplement already available GCase or replace it to break down accumulated glucocerebrosides in different organs, thereby reducing the symptoms of type 1 Gaucher disease.
VPRIV in clinical trials
Experiments conducted in a mouse model of Gaucher disease showed that VPRIV has similar biochemical and safety/efficacy properties as Cerezyme (imiglucerase), another enzyme replacement therapy for Gaucher disease.
A Phase 3 clinical trial (NCT00553631) to compare VPRIV with Cerezyme was completed in 2009. The results showed a rise in hemoglobin levels, an increase in platelet counts, and a reduction in spleen and liver volumes after nine months of treatment.
A Phase 1/2 open-label clinical trial (NCT00391625) to evaluate the long-term safety of VPRIV in patients with type 1 Gaucher disease was completed in 2011. A normalization in liver and spleen volumes was observed with no treatment-related serious side effects over a 48-month period.
A Phase 3 extension trial (NCT00635427) to study the long-term safety of alternate-week dosing of VPRIV was completed in 2012. The results showed the treatment was safe and well-tolerated by patients, and demonstrated a satisfactory clinical response.
VPRIV is available in single-use vials of 200 and 400 units intended for intravenous infusion. The medicine does not contain preservatives, and is in powder form, so it requires reconstitution and dilution.
The most commonly observed side effects in patients treated with VPRIV in clinical studies is hypersensitivity reactions, including headache, dizziness, low/high blood pressure, nausea, tiredness/weakness, and fever. The less common side effects include bone pain, tachycardia, rash, and high blood pressure. VPRIV also carries the risk of an immune reaction.
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