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Results For "rare-disease"

212 News Found

FDA grants Orphan Drug Designation for Tinostamustine in malignant glioma
Drug Approval | November 12, 2025

FDA grants Orphan Drug Designation for Tinostamustine in malignant glioma

Tinostamustine is an investigational drug with a potential first-in-class mechanism combining bifunctional alkylating activity and pan histone deacetylase (HDAC) inhibition


Zydus receives FDA’s ODD for Desidustat for the treatment of beta-thalassemia
Drug Approval | November 06, 2025

Zydus receives FDA’s ODD for Desidustat for the treatment of beta-thalassemia

Orphan drug designation (ODD) by the USFDA for Desidustat, provides eligibility for a potential seven-year marketing exclusivity subject to the USFDA approval


EU approves Alexion’s Koselugo to treat adults with neurofibromatosis type 1 tumours
Clinical Trials | October 31, 2025

EU approves Alexion’s Koselugo to treat adults with neurofibromatosis type 1 tumours

Approval based on pivotal KOMET Phase III trial demonstrating significant tumour reduction in adults with NF1


Alexion highlights breakthrough C5 inhibition advances in gMG at 2025 AANEM and MGFA scientific sessions
Clinical Trials | October 31, 2025

Alexion highlights breakthrough C5 inhibition advances in gMG at 2025 AANEM and MGFA scientific sessions

New Phase III data on gefurulimab highlight potential as a self-administered treatment option; real-world evidence reinforces clinical benefits of Ultomiris and Soliris


Sanofi's Q3 profit dips marginally as Dupixent sales top €4 billion
News | October 27, 2025

Sanofi's Q3 profit dips marginally as Dupixent sales top €4 billion

The company's net income attributable to equity holders dropped to €2.80 billion from €2.82 billion last year


Novo Nordisk acquires Omeros’ MASP-3 inhibitor Zaltenibart for up to US$ 340 million
News | October 17, 2025

Novo Nordisk acquires Omeros’ MASP-3 inhibitor Zaltenibart for up to US$ 340 million

Zaltenibart offers best-in-class potential across multiple rare blood and kidney disorders, enhancing Novo Nordisk’s rare disease portfolio


Delhi High Court refuses to restrain Natco from manufacturing spinal muscular atrophy drug
News | October 10, 2025

Delhi High Court refuses to restrain Natco from manufacturing spinal muscular atrophy drug

The Court dismissed Swiss drugmaker Roche’s plea for interim injunction


India invites applications under PRIP scheme to drive pharma-medtech innovation
R&D | October 06, 2025

India invites applications under PRIP scheme to drive pharma-medtech innovation

Early stage projects of up to Rs. 9 crore eligible for up to Rs. 5 crore assistance and later stage projects of up to Rs. 285 crore eligible for up to Rs. 100 crore assistance