The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation ...

Ariceum Therapeutics, a private biotech company based in Berlin, has announced that the US Food and Drug Administration (FDA) ...

The "Strategic Intelligence: Orphan Designated Drugs" report has been added to ResearchAndMarkets.com's offering.This report consolidates the latest thinking and forecasts around how the healthcare, ...

Ariceum Therapeutics’ proprietary radiopharmaceutical cancer therapy, 225Ac-SSO110 receives US FDA orphan drug designation: Berlin, Germany Friday, February 7, 2025, 11:00 Hrs [ ...

Ariceum Therapeutics (Ariceum), a private biotech company developing radiopharmaceutical products for the diagnosis and treatment of certain hard-to-treat cancers, today announced that the U.S. Food ...

A new drug reimbursement principle is seen as a significant departure from value-based pricing. Despite concerns, the new pricing system could increase the availability of some orphan drugs by 35 per ...

On January 7, 2025, the FDA granted priority review to the new drug application for sunvozertinib (DZD9008), an oral EGFR ...

Cumberland Pharmaceuticals' Phase 2 trial shows ifetroban improves heart function in Duchenne muscular dystrophy patients.

Neurizon Therapeutics has achieved a key milestone with a patent granted in the US for its lead drug candidate NUZ-001 to ...

In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed ...

Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that safety and efficacy ...

Q3 2025 Management View CEO Ken d'Entremont highlighted the successful FDA approval of GRAFAPEX, the branded name for treosulfan for injection in the U.S., which holds Orphan Drug Designation, ...