FDA to Review Pharming’s Application for Ruconest as HAE Swelling Prevention

The U.S. Food and Drug Administration (FDA) has agreed to review Pharming’s application to get Ruconest (conestat alfa) approved for the routine prevention of swelling attacks caused by hereditary angioedema (HAE).

The therapy already is approved for the acute treatment of swelling attacks in adults and adolescents with HAE. Pharming, however, believes the treatment can add additional benefits to HAE patients by preventing the attacks in the first place.

If approved, Ruconest would become the first treatment of its kind approved for both prevention and acute treatment of HAE.

The FDA is expected to deliver a decision on the application by Sept. 21, 2018.

Preventing swelling in HAE is likely to have an immense impact on the lives of HAE patients. If swelling occurs in the throat, nose, or tongue, patients can have difficulty breathing — triggering a potentially fatal situation.

But swelling of skin in the face or other body parts is both disfiguring and painful, researchers said. Swelling of inner organs also causes severe pain, nausea, vomiting, and diarrhea. The disease causes many HAE patients to withdraw from social activities.

Ruconest is a lab-made version of an enzyme that is found naturally in the human body. Called “C1 esterase inhibitor,” it acts by blocking proteins involved in immune and inflammatory processes. Patients with HAE lack natural C1 esterase inhibitor because of a gene mutation.

When the enzyme is lacking, immune and blood flow processes may become excessively activated — causing the soft tissue swelling.

The FDA will base its decision on data from two Phase 2 trials of Ruconest.

The first study (NCT00851409) included 25 patients who received weekly intravenous injections over eight weeks in an open-label fashion.

In the second trial (NCT02247739), researchers compared Ruconest to a placebo in 32 HAE patients. Results, published in the journal The Lancet, showed that once or twice weekly administration of the treatment reduced the number of attacks over four weeks.

The treatment also was safe, with headaches and common colds being the most common adverse events.

Ruconest also was studied in a Phase 2 trial (NCT01359969) in children, aged two to 13, with HAE. Results, so far, have been encouraging. This trial continues to recruit participants. For more information, including locations and contact detail, see the study’s registration page here.