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Wednesday 29 April 2026

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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.

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Chiesi signs $1.9bn deal to buy KalVista and its HAE drug

Chiesi signs $1.9bn deal to buy KalVista and its HAE drug

Chiesi has boosted its rare disease business with an agreement to buy KalVista and Ekterly, its oral drug for treating attacks in rare disease HAE.

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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