X
Final NICE rejection for Biomarin’s childhood rare disease drug
https://pharmaphorum.com/news/final-nice-rejection-for-biomarins-childhood-rare-disease-drug/
BioMarin
FDA approves new treatment for rare genetic disease PKU
Novel enzyme therapy will treat adults with PKU who have uncontrolled blood phenylalanine concentrations with current treatment.
NICE rejects Biomarin’s £520K per year rare disease drug
Company will negotiate access scheme
New NICE rules could deny drugs to rare disease patients
Patients could be denied treatments, says BIA
1 2