Скачать или смотреть Rare Diseases Update – 29 Jan 2026: EU approves C3G treatment, FDA Orphan Designation for MF & More

This biweekly video recap in the rare diseases space covers significant regulatory approvals, clinical trial advancements, new partnerships, and other key developments. It provides a comprehensive overview of the most relevant progress in the field.

🔬 Key highlights:

🧬 Sobi: European Commission approval for Aspaveli® (pegcetacoplan) for C3G and primary IC-MPGN

💊 Atossa Therapeutics: FDA Orphan Drug Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy

🧪 Tanabe Pharma: Positive results for MT-7117 in EPP and XLP
🍁 Recordati Rare Diseases: Isturisa® now available in Canada for Cushing’s disease

🇪🇺 IntraBio: European Commission approval for AQNEURSA® for Niemann-Pick Type C Disease

🔬 Quoin Pharmaceuticals: Submits application for Orphan Drug Designation for QRX003 in Netherton Syndrome

🧬 Mirum Pharmaceuticals: Completes acquisition of Bluejay Therapeutics

💉 Opna Bio: Receives FDA Orphan Drug Designation for Zavabresib in Myelofibrosis

Chapters:
0:00 Introduction
0:08 Sobi: Receives European Commission approval for Aspaveli® (pegcetacoplan) for C3G and primary IC-MPGN
0:39 Atossa Therapeutics: Receives FDA Orphan Drug Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy
1:07 Tanabe Pharma: Announces positive results for MT-7117 in Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP)
1:35 Recordati Rare Diseases: Isturisa® now available in Canada for adults with Cushing’s disease
1:59 IntraBio: Receives European Commission approval for AQNEURSA® for Niemann-Pick Type C Disease
2:21 Quoin Pharmaceuticals: Submits application for Orphan Drug Designation for QRX003 in Netherton Syndrome
2:45 Mirum Pharmaceuticals: Completes acquisition of Bluejay Therapeutics, expanding rare disease portfolio
3:11 Opna Bio: Receives FDA Orphan Drug Designation for Zavabresib in Myelofibrosis
3:39 How to reach us

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