Latest News for: orphan drug

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Evaluate Releases 2026 Orphan Drug Report

Pharmiweb 17 Mar 2026
The top eight orphan drugs will each sell over $6 billion in 2032, with the sales of today’s orphan drug pipeline candidates collectively forecasted to top $100 billion in 2032 The top selling orphan ...
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Aminex Therapeutics Receives Second FDA Orphan Drug Designation for AMXT 1501 for Malignant Glioma Including ...

The Facts 12 Mar 2026
- Designation Underscores the Significant Unmet Medical Need as AMXT 1501 Advances in Two Active National Clinical Trials — Including a Pediatric Study Now Enrolling Children with DIPG ....
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Aminex Therapeutics Receives Second FDA Orphan Drug Designation for AMXT 1501 for Malignant Glioma Including DIPG - a Highly Aggressive Childhood Brain Cancer

PR Newswire 12 Mar 2026
Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AMXT 1501 in combination with difluoromethylornithine (DFMO) for the treatment of malignant glioma, including diffuse intrinsic pontine glioma (DIPG).
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Shilpa Biologicals and mAbTree Biologics Secure Orphan Drug Designation from U.S. FDA

Pharmiweb 12 Mar 2026
Securing Orphan Drug Designation recognizes both the seriousness of ET and PV and the potential of this first-in-class, immunology-driven biologic to modify disease progression rather than only managing symptoms.
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GENFIT’s FDA Orphan Drug Designation for G1090N Opens a New Front in the Race to ...

GetNews 11 Mar 2026
"FDA Orphan Drug Designation for GENFIT's G1090N" ... GNFT) announced that the FDA granted Orphan Drug Designation (ODD) to G1090N, a proprietary reformulation of nitazoxanide (NTZ), for the treatment of ACLF.
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Eledon Pharmaceuticals Announces Orphan Drug Designation Granted to Tegoprubart for the Prevention of Allograft Rejection ...

Nasdaq Globe Newswire 10 Mar 2026
Eledon Pharmaceuticals Announces Orphan Drug Designation Granted to Tegoprubart for the Prevention of Allograft Rejection in Liver Transplantation ... .
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Sanofi’s rilzabrutinib earns orphan drug designation in Japan for IgG4-related disease (Form 6-K) (Sanofi SA)

Public Technologies 10 Mar 2026
Sanofi's rilzabrutinib earns orphan drug designation in Japan for IgG4-related disease • ... Third global orphan drug designation for rilzabrutinib in IgG4-RD, underpinning Sanofi's commitment to rare immune-mediated diseases.
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GENFIT Receives FDA Orphan Drug Designation for NTZ for the treatment of ACLF

Nasdaq Globe Newswire 09 Mar 2026
Food and Drug Administration (FDA) has granted ODD to NTZ (nitazoxanide), its investigational ...
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Global Orphan Drugs Market is Anticipated to Cross USD 350 Billion by 2032 | DelveInsight

PR Newswire 04 Mar 2026
... current and forecast market analysis, individual leading orphan drugs companies' market shares, challenges, orphan drugs market drivers, barriers, trends, and key orphan drugs companies in the market.
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Press Release: Sanofi’s rilzabrutinib earns orphan drug designation in Japan for IgG4-related disease

Nasdaq Globe Newswire 02 Mar 2026
Sanofi’s rilzabrutinib earns orphan drug designation in Japan for IgG4-related disease ... .
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Press Release: Sanofi’s rilzabrutinib earns orphan drug designation in Japan for IgG4-related disease (Sanofi SA)

Public Technologies 02 Mar 2026
Sanofi's rilzabrutinib earns orphan drug designation in Japan for IgG4-related disease Designation based on positive data from a phase 2 study of rilzabrutinib in IgG4-RD Third global orphan drug ...
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Soligenix Receives Positive Opinion from the European Medicines Agency on the Request for Orphan Drug Designation for SGX945 for the Treatment of Behçet's Disease

PR Newswire 26 Feb 2026
... Drug Administration (FDA) for the treatment of Behçet's Disease. Orphan drug designation by the EMA provides a 10-year period of marketing exclusivity in the European Union (EU) after product approval.
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Pain project ACD440 granted orphan drug status in the EU

Pharmiweb 24 Feb 2026
The compound received orphan drug designation from the US Food and Drug Administration (FDA) in 2025 ... Orphan drug status entails a number of ... Also, the price of orphan drugs in general is very high, e.g.
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Tessera Therapeutics Receives U.S. FDA Fast Track and Orphan Drug Designations for its Lead In ...

Nasdaq Globe Newswire 23 Feb 2026
Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to TSRA-196, ...
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PureTech Announces Orphan Drug Designations Granted by the U.S. Food and Drug Administration and European ...

Business Wire 20 Feb 2026
BOSTON--(BUSINESS WIRE)--PureTech Announces Orphan Drug Designations Granted by the U.S. Food and Drug Administration and European Commission for Deupirfenidone in IPF ... .

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