Regulatory Affairs Professionals Society

Study: FDA approves more orphan drugs with specific cancer, pediatric indications over EMA

The US Food and Drug Administration (FDA) is more likely to approve drugs with an orphan designation that were indicated for ...
Miami Herald

FDA Approves Orphan-Drug Designation for Jaguar Health’s Crofelemer for Treatment of Diarrhea in Cholera

Crofelemer previously granted orphan-drug designation by the FDA and the European Medicines Agency (EMA) for both short bowel syndrome and microvillus inclusion disease SAN FRANCISCO, CA / ACCESSWIRE ...
Nasdaq

NurExone Biologic Secures EMA Orphan Status for ExoPTEN in Spinal Cord Injury, Accelerating Pathway to European Markets

TORONTO and HAIFA, Israel, Nov. 13, 2024 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (OTCQB: NRXBF) (Germany: J90) ("NurExone" or the "Company"), a biopharmaceutical company developing ...
RAPS

EU Court Rules on Orphan Product Exclusivity

A European Court has issued a ruling in an unlikely scenario dealing with overlapping periods of orphan product market exclusivity. In its ruling, which has the potential to influence how companies ...
Hosted on MSN

Sanofi receives orphan status from EMA for rilzabrutinib

Sanofi has received orphan designation from the European Medicines Agency (EMA) for rilzabrutinib, a reversible covalent Bruton’s tyrosine kinase (BTK) inhibitor, to treat immunoglobulin G4 ...
JD Supra

The European Commission Proposes First Major Overhaul of the EU Medicines Regulatory Framework in 20 Years: Orphan Medicines

We recently published an alert in relation to the European Commission’s legislative proposals to replace the current EU regulatory framework for all medicines (including those for rare diseases and ...
JD Supra

FDA Doubles Down on Its Pre-Catalyst Stance on Orphan Drug Exclusivity

Under the Orphan Drug Act, the FDA may grant orphan drug designation to a drug or biologic intended to treat a rare disease or condition, defined as a patient population of fewer than 200,000 ...
EurekAlert!

Gate2Brain receives EMA orphan drug designation for its pediatric cancer treatment

The IRB Barcelona spin-off Gate2Brain, a cutting-edge technology platform focused on enhancing drug delivery to the brain, has reached a major milestone in the fight against pediatric brain tumors by ...
Nasdaq

Candel Therapeutics Receives EMA Orphan Designation for CAN-2409 in Pancreatic Cancer Treatment

Candel Therapeutics, Inc. has announced that the European Medicines Agency (EMA) has granted Orphan Designation for its investigational treatment CAN-2409 for pancreatic cancer, complementing its ...
Health Affairs

Orphan Drug Label Expansions: Analysis Of Subsequent Rare And Common Indication Approvals

The Orphan Drug Act of 1983 was enacted to provide financial incentives to stimulate drug development for rare diseases. In recent years, concerns have been raised regarding these orphan drugs, ...
Yahoo Finance

Medicus Pharma files Orphan Drug application for SkinJect in rare skin Cancer indication

Medicus Pharma CEO Dr Raza Bokhari joined Steve Darling from Proactive to announce that the company has submitted an Orphan Drug Designation application to the U.S. Food and Drug Administration for ...