Shire Submits sBLA for HAE Therapy to Include Pediatric Indication

The FDA has accepted a supplemental Biologics License Application (sBLA) for Shire’s Cinryze, a therapy that treats recurring swelling attacks in hereditary angioedema (HAE), to expand the already approved indication to include children 6 years and older.

HAE is a rare, immune disorder in which patients experience recurrent episodes of severe swelling on the limbs, face, intestinal tract, and airway. Serious episodes can restrict breathing and cause obstruction of the airway, while also developing a rash on the patient’s skin.

Typically, attacks occur once every 1 to 2 weeks for a period of 3 to 4 days. Nonetheless, the frequency and severity of swelling attacks depends on the individual.

The condition commonly begins during childhood and worsens as the patient matures. Some HAE patients can experience abdominal pain from the accumulation of fluid in the abdomen, and facial puffiness. The goal of treatment is to prevent such attacks and make patients more comfortable when attacks do arise.

About 1 in 10,000 to 1 in 50,000 patients live with HAE worldwide.

Cinryze is a routine prophylaxis containing the active substance C1-Esterase Inhibitor (C1-INH) that decreases the amount of swelling by replenishing the deficient protein while regulating the levels of bradykinin released during an attack.

In 2008, the FDA approved Cinryze to prevent attacks in adolescents and adults diagnosed with HAE. The sBLA for Cinryze was granted based on data from 2 open-label studies and two pediatric clinical studies.

Cinryze has also received Priority Review designation which accelerates the review target to 8 months instead of 12 months. This is based on the safety and efficacy of the treatment displayed in previous clinical trials.

Currently, Shire is the only HAE drug developer committed to extending their prophylactic treatment to children through their completion of pediatric studies. If approved by the FDA, Cinryze will be the only available C1-INH therapy used to prevent HAE attacks from childhood to adulthood.

"Adults and adolescents living with HAE have used CINRYZE to help reduce the frequency and severity of attacks for nearly a decade," said Jennifer Schranz, Global Development Lead, HAE, Shire in a press release. "Shire is committed to studying the safety and efficacy of our HAE therapies in children aged 6 years and older because we understand the importance of this work to families in the HAE community. We look forward to working closely with the FDA in the coming months on this important review."

An FDA decision on the proposed expansion is expected by June 20,2018.