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EU orphan designations for OPKO's clotting Factor VIIa-CTP

EU orphan designations for OPKO's clotting Factor VIIa-CTP

10-07-2014

The European Committee for Orphan Medicinal Products (COMP) gave a positive opinion recommending the…

EuropeFactor VIIa-CTPHematologyOPKO HealthPharmaceuticalRare diseasesRegulation

Added indications recommended by EMA/CHMP

Added indications recommended by EMA/CHMP

27-06-2014

Along with backing for approval for six news drugs issued at the late June meeting of the European Medicines…

AvastinBayerBristol-Myers SquibbCardio-vascularEliquisEuropeEyleaKalydecoOncologyOphthalmicsPfizerPharmaceuticalRare diseasesRegulationRocheVertex Pharmaceuticals

EU approval for Janssen's Sylvant to treat patients with MCD

EU approval for Janssen's Sylvant to treat patients with MCD

08-06-2014

The European Commission (EC) has approved the use of Janssen-Cilag International's Sylvant (siltuximab)…

EuropeJanssen-CilagJohnson & JohnsonPharmaceuticalRare diseasesRegulationSylvant

CHMP changes mind on PTC Therapeutics’ Duchenne drug Translarna, giving a positive opinion

25-05-2014

In something of a surprise for PTC Therapeutics and observers, the European Medicines Agency’s advisory…

BiotechnologyEuropePTC TherapeuticsRare diseasesRegulationTranslarna

Santhera re-files for EU approval of Raxone in the treatment of LHON

07-05-2014

Swiss drugmaker Santhera Pharmaceuticals has re-filed its Marketing Authorization Application for Raxone…

EuropeOphthalmicsPharmaceuticalRare diseasesRaxoneSanthera Pharmaceuticals

EMA and Australia’s TGA collaborate in the area of orphan medicines

EMA and Australia’s TGA collaborate in the area of orphan medicines

07-04-2014

During a meeting last week at the European Medicines Agency, the EMA’s executive director, Guido Rasi,…

AustraliaEuropePharmaceuticalRare diseasesRegulation

Jazz Pharma and Gentium debut Defitelio in Europe

31-03-2014

Ireland-headquartered Jazz Pharmaceuticals and its Italian subsidiary Gentium have initiated the European…

AustriaDefitelioEuropeGentiumGermanyJazz PharmaceuticalsMarkets & MarketingPharmaceuticalRare diseases

EMA/CHMP recommends 12 new orphan medicines over the past year

26-02-2014

Over the past 12 months, a total of 12 medicines for the treatment of rare diseases were recommended…

EuropePharmaceuticalRare diseasesRegulation

Positive CHMP opinion for BioMarin’s Vimizim for Morquio A syndrome

Positive CHMP opinion for BioMarin’s Vimizim for Morquio A syndrome

21-02-2014

BioMarin Pharmaceutical says that the European Medicines Agency’s Committee for Medicinal Products…

BioMarin PharmaceuticalEuropePharmaceuticalRare diseasesRegulationVimizim

Eisai’s amatuximab gains orphan status in EU for malignant mesothelioma

Eisai’s amatuximab gains orphan status in EU for malignant mesothelioma

22-01-2014

The investigational monoclonal antibody amatuximab (development code: MORAb-009), has today been granted…

amatuximabEisaiEuropePharmaceuticalRare diseasesRegulation

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