With FDA nod for a Novartis castoff, Pharming is set to market a second rare disease drug

With an infrastructure in place to market its lone commercial product Ruconest, it made sense in 2019 for Pharming Group to scoop up another rare disease candidate in the same arena.

Four years later, Pharming’s $20 million purchase of leniolisib from Novartis is set to bear dividends.

On Friday, the FDA signed off (PDF) on Joenja for patients with phosphoinositide 3-kinase delta syndrome (APDS). It becomes the first disease-modifying treatment for the condition, which was discovered just 10 years ago. Patients with APDS have historically been given antibiotics, immunosuppressants and immunoglobulin replacement drugs to deal with the symptoms.   

The endorsement covers patients for the genetic disease 12 and older. The small molecule is designed to block the PI3K-delta protein, which inhibits signaling pathways that lead to dysregulation of the B and T immune cells.  

Pharming, which is based in Leiden, Netherlands, reported sales of $206 million for protein deficiency angioedema (HAE) drug Ruconest last year. It has 54 sales reps In the United States who will now begin pitching Joenja, ahead of its launch next month.

“It’s not our first rodeo,” Sijmen de Vries, M.D., Pharming’s CEO said in a conference call on Monday. “We have very experienced and dedicated commercial and medical teams that are in place.”

Pharming has priced Joenja at $750 per tablet, which are taken twice daily, coming to roughly $547,500 annually.

The company believes there are 1,500 or more APDS patients across the U.S., Canada, European Union and Japan, including 500 who have been identified, with nearly 200 of them in the U.S. A genetic test is required to identify those with APDS.

“Patients are easily misdiagnosed and they’re often hiding in plain sight,” Stephen Toor, Pharming’s chief commercial officer said, adding that the company will work to educate physicians about APDS. Family mapping is an important component in the identification of patients as each child born to a parent with the condition has a 50-50 chance of acquiring it, Toor added.

With the approval, Pharming is on the hook for a $10.5 million milestone payment to Novartis and another unidentified party. As commercial milestones are reached, Novartis could receive up to $190 million more in payments. Novartis also will receive tiered royalties from sales, ranging from a percentage in the low-to-high teens.

The FDA’s approval comes under priority review and was based on results of a phase 2/3 trial of 31 patients which met its primary co-endpoints, showing Joenja reduced lymph node size and increased naïve B cells compared with placebo.

Those with APDS are left with fewer white blood cells that fight pathogens and can become more susceptible to infections in the lungs, sinuses and ears. It can also lead to swelling of the lymph nodes, spleen and tonsils, creating obstructions.

The protein deficiency also makes patients more prone to blood cancers, which can be treated with chemotherapy and stem cell transplants. But those can increase the risk of new complications.

Pharming has filed with regulators in Europe and expects marking authorization in the second half of this year. The company also is exploring the licensing of other rare disease assets.

“Because we have a very scalable commercial operation and because we have a strong track record in the development and commercialization of rare disease assets, we continue our hunt to get advanced projects—at an advanced clinical stage to add to our pipeline,” de Vries said.