MarketPulse

enzyme replacement therapy

4 articles
BenzingaBenzinga··Vandana Singh

AstraZeneca Splits on HPP Drug as COPD Candidate Advances

AstraZeneca's efzimfotase alfa succeeds in pediatric HPP trials but fails in adults. Meanwhile, tozorakimab meets COPD endpoints across smoker populations.
AZNPhase 3 trialsCOPD
GlobeNewswire Inc.GlobeNewswire Inc.··Researchandmarkets.Com

Lamzede Market Poised for Expansion as Rare Disease Treatments Gain Momentum

Lamzede market expands globally, led by Europe with fastest growth in Asia-Pacific, driven by rare disease diagnostic advances and enzyme replacement therapy adoption.
REGNpersonalized medicinebiotechnology
GlobeNewswire Inc.GlobeNewswire Inc.··Chiesi Global Rare Diseases / Protalix Biotherapeutics

Elfabrio Wins EU Approval for Less-Frequent Dosing, Triggering $25M Payment

European Commission approves every-4-weeks dosing regimen for Elfabrio, reducing patient treatment burden and triggering $25 million milestone payment to Protalix from Chiesi.
PLXrare diseaseEuropean Commission approval
GlobeNewswire Inc.GlobeNewswire Inc.··Researchandmarkets.Com

Skeletal Dysplasia Market Set to Surge 54% to $5.37B by 2032

Global skeletal dysplasia market projected to grow from $3.48B (2026) to $5.37B (2032) at 7.42% CAGR, driven by diagnostic advances and innovative therapies.
PFENVSRAREREGNBMRNrare diseasebiologic therapy