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Home»Posts tagged with»orphan drug designation

Emalex Biosciences advances Tourette syndrome strategy as ecopipam delays relapse in Phase 3 trial

By Pallavi Madhiraju on May 27, 2026   Pharma & Biotech  

Emalex Biosciences advances Tourette syndrome strategy as ecopipam delays relapse in Phase 3 trial

D1 receptor targeting may change Tourette syndrome care. Ecopipam’s Phase 3 data now face the bigger test: regulation and adoption.

Is BRC Therapeutics opening a new regulatory lane for cannabinoid-based CRPS therapies?

By Pallavi Madhiraju on May 1, 2026   Pharma & Biotech  

Is BRC Therapeutics opening a new regulatory lane for cannabinoid-based CRPS therapies?

CRPS has no approved U.S. therapy. BRC-002 now has an FDA path, but proof of safety, efficacy, and dosing discipline still lies ahead.

Is Can-Fite BioPharma Ltd. building a differentiated niche in late-line pancreatic cancer therapy

By Soujanya Ravi on April 30, 2026   Pharma & Biotech  

Is Can-Fite BioPharma Ltd. building a differentiated niche in late-line pancreatic cancer therapy

Is Can-Fite BioPharma Ltd. carving a niche in pancreatic cancer? Explore how namodenoson’s Phase 2a data could reshape late-line treatment strategies.

What Orion Corporation’s FDA orphan designation for ODM-212 means for mesothelioma treatment innovation

By Soujanya Ravi on April 20, 2026   Pharma & Biotech  

What Orion Corporation’s FDA orphan designation for ODM-212 means for mesothelioma treatment innovation

Discover how Orion Corporation’s ODM-212 orphan drug status could reshape mesothelioma treatment and drive innovation in Hippo pathway targeting.

What Medicus Pharma Ltd.’s SkinJect application signals for Hedgehog pathway-driven cancers

By Soujanya Ravi on April 17, 2026   Pharma & Biotech  

What Medicus Pharma Ltd.’s SkinJect application signals for Hedgehog pathway-driven cancers

Discover how SkinJect’s orphan drug bid could reshape Hedgehog-driven cancer treatment. Read what it means for clinicians, regulators, and investors.

Could Ampligen become a late-stage pancreatic cancer breakthrough after AIM ImmunoTech’s latest clinical push?

By Soujanya Ravi on April 16, 2026   Pharma & Biotech  

Could Ampligen become a late-stage pancreatic cancer breakthrough after AIM ImmunoTech’s latest clinical push?

Can Ampligen’s Phase 3 push reshape pancreatic cancer treatment? Read how AIM ImmunoTech’s latest clinical progress could change the oncology outlook.

Can Immutep Limited convert orphan designation into a credible late-stage oncology program?

By Soujanya Ravi on April 15, 2026   Pharma & Biotech  

Can Immutep Limited convert orphan designation into a credible late-stage oncology program?

FDA orphan drug designation gives Immutep Limited a new sarcoma pathway. Find out why eftilagimod alfa could reshape its oncology strategy.

Can REYOBIQ improve Plus Therapeutics’ path in pediatric high-grade glioma and ependymoma?

By Pallavi Madhiraju on April 11, 2026   Pharma & Biotech  

Can REYOBIQ improve Plus Therapeutics’ path in pediatric high-grade glioma and ependymoma?

Plus Therapeutics won orphan drug designation for REYOBIQ in pediatric malignant gliomas. Read what it changes for CNS cancer development.

How REYOBIQ’s latest regulatory milestone may reshape the pediatric neuro-oncology landscape

By Soujanya Ravi on April 8, 2026   Uncategorized  

How REYOBIQ’s latest regulatory milestone may reshape the pediatric neuro-oncology landscape

Plus Therapeutics secures orphan designation for REYOBIQ in pediatric malignant gliomas. Read what this means for trials, exclusivity, and market strategy.

Can Abbisko Therapeutics turn irpagratinib into the first approved FGFR4 therapy for hepatocellular carcinoma?

By Pallavi Madhiraju on April 5, 2026   Pharma & Biotech  

Can Abbisko Therapeutics turn irpagratinib into the first approved FGFR4 therapy for hepatocellular carcinoma?

Abbisko Therapeutics won EMA orphan drug designation for irpagratinib in liver cancer. Read what this changes for FGFR4-targeted therapy.

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