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ENERGIZE-T trial

PharmaDeviceNews ENERGIZE-T trial

Representative image: A haematology consultation illustrates how PYRUKYND’s European Union approval for adults with thalassaemia could shift attention toward oral rare blood disease treatments, patient access and reimbursement across Europe.

Pharma & Biotech

Could Agios and Avanzanite turn PYRUKYND into Europe’s next rare blood disease launch test?

by Pallavi Madhiraju
May 23, 2026
0 Comments

PYRUKYND has EU approval in thalassaemia. Now Agios and Avanzanite face Europe’s harder test: access, uptake and reimbursement.

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Niagen Bioscience’s NB4168 has secured U.S. FDA Rare Pediatric Disease and European orphan designations as the company advances the experimental ataxia telangiectasia therapy toward human trials. Representative image.

Pharma & Biotech

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