US FDA approves drug to treat genetic disorder

Joenja is expected to launch in the US in early April and will be available for shipment in mid-April, company says

By
Reuters
Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, US. — Reuters/File
Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, US. — Reuters/File

The US Food and Drug Administration (FDA) on Friday approved Pharming Group's drug to treat a rare genetic disorder that leads to a weakened immune system, the Dutch company said.

Leniolisib, to be sold under the brand name Joenja, becomes the first approved drug in the United States to treat activated phosphoinositide 3-kinase delta syndrome (APDS), a primary immunodeficiency that affects about 1 to 2 people in a million.

Pharming's US-listed shares rose 22% to $13.75.

Joenja is expected to launch in the US in early April and will be available for shipment in mid-April, the company said.

The approval comes a month after the European health regulator shifted the accelerated review of leniolisib to a standard one, citing the need for the company to submit additional data from an extension study that was conducted the post-interim analysis.

APDS causes a lack of functioning immune cells, B cells and T cells, which makes it difficult for people with this disorder to fight off bacterial and viral infections. It also exacerbates the risk of permanent lung damage and lymphoma over time.

Pharming, which acquired global rights to Joenja from Novartis in 2019, declined to comment on the pricing and said it would provide more details on Monday.

At least two analysts expected the drug to be priced at as much as $400,000.

Oppenheimer analyst Hartaj Singh expected Joenja to be priced between $250,000 and $400,000, and forecast peak sales of $200 million to $300 million, ahead of the approval.

The FDA approval was based on data from a late-stage study, which showed the drug helped normalise immune function as measured by a significant increase in the number of immune response-generating B cells and a reduction in the size of lymph nodes.

Joenja is an oral drug that targets and blocks a form of the protein called phosphoinositide 3-kinase delta (P13K).