Shire immunodeficiency drug wins U.S. approval
- The U.S. FDA has approved Shire’s Cuvitrue immunoglobulin for the treatment of primary immunodeficiency, a group of 300+ genetic immune system dysfunctions. This follows approvals in 17 European countries since June 2016.
- Shire plans further submissions worldwide in 2016 and 2017 for Cuvitru, which is a 20% immune globulin solution delivered subcutaneously.
- According to Transparency Market Research, the global primary immunodeficiency diseases market was worth almost $4.4 billion in 2014 and could grow by 6.1% (CAGR) to $7.5 billion by 2023, driven by the emergence of new therapeutics.
Shire claims that Cuvitru can be administered rapidly and at higher volumes than other conventional subcutaneous IG treatments. This means patients may be able to have shorter infusions at fewer sites.
"As the national patient organization dedicated to people with primary immunodeficiency, the Immune Deficiency Foundation is delighted to see new treatment options that can address patients' clinical priorities as well as their personal preferences, particularly in terms of dosing and administration," said Marcia Boyle, president and founder of the Immune Deficiency Foundation.
It’s not all sunshine and roses though – Cuvitru comes with a black box warning of thrombosis, which is common to immune globulin products.
Primary immunodeficiencies are genetic disorders that lead to a range of conditions, from dysfunctional to completely absent immune systems. These leave people vulnerable to life-threatening bacterial, viral and fungal infections. While the individual genetic disorders are rare, primary immunodeficiency overall affects up to six million people worldwide.
Treatment ranges from preventing infection through vaccinations and long-term antibiotics to boosting the immune system with immunoglobulins and stem cell transplants. Gene therapy has been touted as a treatment, or even a cure, for some of the primary immunodeficiencies, by recreating a fully-functioning immune system. Unfortunately, as described in a review in F1000Research, some serious side effects and fatalities showed up in early studies in the 1990s, and that put the brakes on the research. More recently, clinical trials in severe forms of primary immunodeficiencies are looking hopeful, but this will depend on continually improving technologies and long term follow up of treated patients.
- Shire Statement
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