According to a recent story from pm360online.com, the pharmaceutical company Shire announced that the marketing application for its drug product lanadelumab has gained approval from the European Medicines Agency (EMA). Furthermore, the company has also reported that that its New Drug Submission has been adopted with Priority Review by Health Canada for the drug. Lanadelumab is in development for the treatment of patients 12 years and up that have hereditary angiodema.
Hereditary angiodema is a rare genetic disorder in which the patient experiences periodic episodes of dramatic swelling. This swelling can affect many different parts of the body such as digestive tract, arms, legs, face, and airway. In severe cases, swelling can lead to airway obstruction which should be treated as a medical emergency. When the digestive tract is affected, vomiting and abdominal pain may occur. It is caused by a genetic mutation that is either inherited or can occur as a new mutation. When left untreated, attacks can occur every couple of weeks and often last for a few days. With treatment, outcomes are usually good, but there are only a few options available in most countries. To learn more about hereditary angiodema, click here.
So far, there is no treatment available that has been shown to be able to completely prevent swelling attacks from happening, but lanadelumab might have the potential to change the situation. The drug is formulated for administration under the skin. The results of trial studies so far have been favorable for the therapy; a 300mg dose every two weeks was able to reduce the average frequency of swelling episodes by 87 percent. Through the course of the trial a 91 percent rate of reduction was achieved and eight of every ten patients remained free of swelling attacks. Lanadelumab also did not display and severe side effects; the most common side effect was some pain at the site of injection.
The validation of the marketing application will mean that a period of accelerated evaluation for the treatment will begin. Hopefully, if the drug continues to do well in future trials, it will be available soon for public use in the EU.