FDA Requests Another Trial for Pharming's Ruconest Label Expansion

Rejection Thumbs Down

The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL), basically a rejection, to Netherlands-based Pharming Group for its supplemental Biologics License Application (sBLA) for Ruconest. The company was trying to get the drug’s label expanded to include prophylaxis in patients with hereditary angioedema (HAE).

HAE is a rare genetic disorder characterized by spontaneous and recurrent episodes of swelling in various parts of the body. It also affects the airways and internal organs. Edema of the skin can be disfiguring, uncomfortable or painful, but edema of the throat, nose or tongue can be life-threatening. Most HAE patients also have severe abdominal pain, nausea, vomiting and diarrhea from intestinal wall swelling.

“While today’s FDA decision is not what we were anticipating, we look forward to working with the FDA to generate additional clinical data required to enable access for patients to use Ruconest for HAE prophylaxis,” stated Bruno Giannetti, Pharming’s chief operations officer.

The FDA requested Pharming run another clinical trial in response to its CRL. The agency approved Ruconest for acute HAE attacks in adults and adolescents in 2014, and the European Union approved it in 2010 for HAE.

The drug is an injectable plasma-free C1-esterase inhibitor (C1-INH) which helps HAE attacks caused by an insufficient blood protein, C1-INH, or when C1-INH isn’t working the right way.

In January, the FDA indicated Pharming’s filing was enough to review. Other drugs approved for prophylaxis for HAE include Behring’s Haegarda, and Shire’s Takhzyro and Cinryze. Other drugs approved for HAE attacks are CSL Behring’s Berinert, Shire’s Cinryze, Takhzyro and Firazyr, and Dyax’s Kalbitor.

“We see this as a minor setback,” stated Sijmen de Vries, Pharming’s chief executive officer. “Pharming remains committed to serving the HAE community. We will continue and have the resources to develop new innovative and more convenient administration options of Ruconest for acute treatment and prophylaxis of HAE to improve patient care as outlined in our recent capital market briefing in June.”

Although de Vries sounds unconcerned, the investors weren’t as thrilled, company shares dropping 22 percent on the Paris stock exchange after the announcement.

At its six-month financial report in July, the company announced Ruconest was being evaluated in Switzerland in a clinical trial for contrast-induced nephropathy, a head-to-head trial against a competitor in acute attacks of HAE, a trial to treat delayed graft function at the University of Wisconsin. The company also had a Phase I/II trial of rhC1INH in pre-eclampsia to be filed in the fourth quarter and an expected trial filing for recombinant human alphaglucosidase in Pompe disease in early 2019.

“We are delighted with the further progress we have made expanding the reach for Ruconest, allowing more patients to access the clinical benefits of our product,” de Vries stated at the time. “We have continued net profitability in the second quarter of the year, which gives us the confidence and the financial resources to move forward with our new programs. With five studies underway or expected to initiate over the next six months, we anticipate significant strengthening of our pipeline.”

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