Orphan Europe company information, Employees & Contact Information

Biodexa Receives Orphan Drug Designation in Europe for eRapa in FAP Yahoo Finance

SPG601 granted orphan drug status in EU for treating fragile X Fragile X News Today

Cystic Fibrosis & Amyloidosis Orphan Drugs Get European Yes Medscape

Sanfilippo B therapy JR-446 gets orphan drug status in Europe Sanfilippo Syndrome News

Meeting abstracts from the 12th European Conference on Rare Diseases and Orphan Products Orphanet Journal of Rare Diseases

Challenges for orphan medicines entering the European market PMLiVE

FNE Oscar Watch 2026: Hungary Selects Orphan as Oscar Bid Film New Europe

Otsuka Sibeprenlimab Phase 3 Data Show a Statistically Significant and Clinically Meaningful Proteinuria Reduction for the Treatment of Immunoglobulin A Nephropathy (IgAN) Mynewsdesk

EMA recommends NUZ-001 for orphan drug status in Europe ALS News Today

Orphan drug market access in Europe: HTA challenges and the growing potential of RWE Pharmaceutical Technology

Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries Frontiers

Dyne Therapeutics Receives European Medicines Agency (EMA) GlobeNewswire

Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe ScienceDirect.com

New pilot programme to support orphan medical devices European Medicines Agency

Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries Frontiers

Trends in orphan medicinal products approvals in the European Union between 2010–2022 Orphanet Journal of Rare Diseases

Realising the opportunities for growth in the European orphan drug market Pharmaceutical Technology

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks Frontiers

Clinical aspects of reimbursement policies for orphan drugs in Central and Eastern European countries Frontiers

An analysis of orphan medicine expenditure in Europe: is it sustainable? Orphanet Journal of Rare Diseases

Reforming EU and national orphan drug regulations to improve outcomes for patients with rare diseases The BMJ

An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries Orphanet Journal of Rare Diseases

Alex Warren announces 'Little Orphan' EU & UK arena tour - How to get tickets Capital UK

Regulatory Standards in Orphan Medicinal Product Designation in the EU Frontiers

Comparing access to orphan medicinal products in Europe Orphanet Journal of Rare Diseases

Intellia Therapeutics Receives European Union Orphan Drug Designation for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema Intellia Therapeutics

Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment Frontiers

EU Orphan Drug Regulations Haven't “Failed” But Reforms Needed, Says CEO Of European Rare Disease NGO Health Policy Watch

European Commission Grants Ocugen Orphan Medicinal Product Designation for Gene Therapy Product Candidate, OCU400, For the Treatment of Both Retinitis Pigmentosa and Leber Congenital Amaurosis Ocugen

Tackling Salami Slicing and Indication Stacking in Orphan Drug Innovation Incentives Petrie-Flom Center

Intellia Therapeutics’ Investigational CRISPR Treatment NTLA-2001 Receives European Union Orphan Drug Designation for ATTR Amyloidosis Intellia Therapeutics

What will the orphan drug market exclusivity haircut mean for industry? AgencyIQ by POLITICO

The new EU “Pharma Package”: Global (Orphan) Marketing Authorization Crowell & Moring LLP

Sumitomo Pharma America Announces that the European Medicines Agency Has Granted Orphan Drug Designation to Nuvisertib (TP-3654) for the Treatment of Myelofibrosis PR Newswire

The orphan drug pipeline in Europe Nature

Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU BMJ Open

Press Release: Sanofi’s rilzabrutinib earns orphan designation in the EU for IgG4-related disease Yahoo Finance

EMA grants orphan designation to ADX-2191 (Aldeyra Therapeutics) for inherited retinal dystrophies Ophthalmology Times Europe

MDCG guidance tries to reduce burden of bringing orphan devices to EU market Regulatory Affairs Professionals Society | RAPS

Ocugen Announces European Medicines Agency Grants Orphan Medicinal Product Designation for Modifier Gene Therapy Candidate OCU410ST for Treatment of ABCA4-Associated Retinopathies including Stargardt Disease Ocugen

RECORDATI AND HELSINN AGREEMENT FOR THE EXCLUSIVE COMMERCIALIZATION RIGHTS TO LEDAGA® GlobeNewswire

ReAlta Life Sciences Secures European Orphan Drug Status for Graft-vs-Host Disease Treatment Yahoo Finance

Workshop on regulatory support for development of orphan medicines European Medicines Agency

How does HTA for orphan drugs differ across Europe? pharmaphorum

Tempest Receives Orphan Drug Designation from the European GlobeNewswire

New chair elected for EMA’s Orphan Medicinal Products Committee European Medicines Agency

Market access of orphan drugs: one size fits all? Hospital Pharmacy Europe

DYNE-101 gets orphan drug status for DM1 in Europe | ACHIEVE Phase... Muscular Dystrophy News

AOP Orphan announces launch of access program for EGRIFTA® Hospital Pharmacy Europe

KalVista Pharmaceuticals Announces Maintenance of Orphan Designation for Sebetralstat in European Union Yahoo Finance

Regulation of orphan drugs within the EU European Pharmaceutical Review

Hamden biotech’s drug earns ‘orphan’ status in Europe Hartford Business Journal

EU Committee Urges Orphan Drug Status for Clene’s CNM-Au8 for ALS... ALS News Today

Novartis announces European Medicines Agency (EMA) has granted orphan drug designation for iptacopan (LNP023) in IgA nephropathy (IgAN) Novartis

Ipsen to refile Alagille drug after EU blocks orphan status pharmaphorum

A GP's view: Orphan drug costs ‘prohibitive’ BBC

Sobi supports The Rare Project to increase awareness about rare diseases Hospital Pharmacy Europe

Charades, New Europe Board Laszlo Nemes’ ‘Orphan’ Ahead Of June Shoot Deadline

Carbaglu approved for N-acetylglutamate synthase (NAGS) deficiency Medical Professionals Reference

The EU pharmaceutical package: a look at the future orphan legislation Simmons & Simmons

Europe Creates Orphan Works Registry, Copyright ID System; Digitises EU Content Intellectual Property Watch

Ibuprofen in the treatment of neonatal PDA Hospital Pharmacy Europe

NTI gets positive opinion on EU orphan drug application The Australian

Biovitrum to Acquire Swedish Orphan for Over $500M Genetic Engineering and Biotechnology News