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APDS

News

FDA knocks back Pharming's bid for wider Joenja use

FDA knocks back Pharming's bid for wider Joenja use

The FDA has turned down Pharming's bid to broaden the label of Joenja for rare immunodeficiency disorder APDS so it can be used in younger patients.

News

Box and bottle of Joenja

First drug for rare disease APDS cleared for NHS use

Pharming's Joenja has become the first disease-modifying drug that can be used by the NHS to treat APDS, an ultra-rare immunodeficiency disorder.

News

Pharming brings first drug for rare disease APDS to US

Pharming brings first drug for rare disease APDS to US

Joenja will launch in April and could make upwards of $200m a year in sales, say analysts.

Editor's Picks

ESMO26: Tango sees 82% ORR with combo in pancreatic cancer

Roche offers $15bn for strategic-level deal with Hansoh

ESMO26: Moderna, MSD reveal cancer jab data early after leak

Clinical trial setup times fall sharply in UK

Despite US policy changes, drug prices stay stubbornly high

News

Flagship joins three biotechs together to form Etcelera

Flagship joins three biotechs together to form Etcelera

Flagship Pioneering has formed a new AI-focused biotech company, called Etcelera, by combining the platforms of three firms from its portfolio.

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The rights of trademark owners are acknowledged. Website and content copyright © 2009-2026, pharmaphorum media limited or its licensors; all rights reserved. pharmaphorum – an EVERSANA company