Research & Consultant Companies

The European Commission granted orphan designation to magrolimab, a humanised monoclonal antibody targeting CD47, for treating acute myeloid leukaemia (AML). This designation acknowledges the...

Gilead Sciences Ireland UC has successfully obtained orphan designation from the European Commission for magrolimab, a novel treatment targeting myelodysplastic syndromes (MDS). This designation...

The European Medicines Agency (EMA) officially designated a novel therapy for myasthenia gravis as an orphan medicinal product on December 13, 2024. This designation...

A significant advancement in the treatment of acute liver failure has been recognized by the European Commission, granting orphan designation to ornithine phenylacetate. This...

Brainvectis, a French biotechnology company, received orphan designation from the European Commission for its innovative treatment targeting Huntington's disease. This designation, granted on April...

The European Commission has awarded orphan designation to IQVIA RDS Ireland Limited for pelabresib monohydrate, a promising therapeutic agent targeting myelofibrosis. This designation highlights...

Orphix Consulting GmbH withdrew Upifitamab rilsodotin from the EU Register of orphan medicinal products in February 2025. Initially designated on December 9, 2022, this...

Theranexus S.A.S., a pharmaceutical company based in France, secured orphan designation from the European Commission for its innovative treatment, trehalose, targeting neuronal ceroid lipofuscinosis....

The European Commission has officially designated a novel therapeutic protein for the treatment of X-linked hypohidrotic ectodermal dysplasia, also known as Christ-Siemens-Touraine Syndrome. This...

A European regulatory milestone was reached as Sanofi B.V. obtained orphan status for its innovative therapy targeting hyperphenylalaninaemia, signaling a significant advancement for patients...

Mirum Pharmaceuticals International B.V. has achieved a significant milestone as its active substance, volixibat potassium, secures orphan designation from the European Medicines Agency (EMA)...

The European Medicines Agency (EMA) officially designated epertinib as an orphan medicinal product for treating amyotrophic lateral sclerosis (ALS) on February 26, 2025. This...

Sarepta Therapeutics Ireland Limited has obtained orphan designation from the European Medicines Agency (EMA) for its innovative treatment targeting limb-girdle muscular dystrophy (LGMD). This...

The European Commission has officially granted orphan status to venetoclax, a promising treatment developed by AbbVie Deutschland GmbH & Co. KG, for patients battling...

The European Medicines Agency (EMA) has officially designated a novel treatment as an orphan medicinal product for Olmsted syndrome as of February 26, 2025....