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SOBI restructures Kineret and Kepivance deals


Swedish Orphan Biovitrum (STO: SOBI) said this morning that it has acquired the full rights to develop…

AmgenAnti-Arthritics/RheumaticsBiotechnologyKepivanceKineretLicensingOncologyPharmaceuticalRare diseasesSwedish Orphan Biovitrum

Kyowa Hakko Kirin collaborates with Ultragenyx on KRN23


Japanese mid-sized drugmaker Kyowa Hakko Kirin (TYO: 4151) has entered into a collaboration and license…

BiotechnologyKRN23Kyowa Hakko KirinLicensingPharmaceuticalRare diseasesResearchUltragenyx

Chiesi expands rare disease portfolio with acquisition


Privately-owned Italian drugmaker Chiesi Group has signed, through its UK affiliate, a definitive agreement…

BiotechnologyChiesi FarmaceuticiLamazymMergers & AcquisitionsPharmaceuticalRare diseasesZymenex

Shire and Santaris extend strategic alliance


Danish privately held clinical-stage biopharmaceutical company Santaris Pharma says that its long term…

BiotechnologyLicensingPharmaceuticalRare diseasesResearchSantaris PharmaShire

New Zealand to widen access for type 1 and type 3 Gaucher disease drug


New Zealand's Pharmaceutical Management Agency PHARMAC has announced the approval of a proposal to widen…

Asia-PacificBiotechnologyCerezymeGenzymePharmaceuticalRare diseasesRegulationSanofi

Aegerion gains European approval for rare disease drug


The European Commission has approved US biopharma company Aegerion Pharmaceuticals' (Nasdaq: AEGR) Lojuxta…

Aegerion PharmaceuticalsBiotechnologyCardio-vascularEuropeJuxtapidKynamroLojuxtalomitapidePharmaceuticalRare diseasesRegulation

UK's NICE calls for more data to consider Roche drug for a type of vasculitis


UK drug watchdog the National Institute for Health and Care Excellence (NICE) has today (July 23) published…

BiotechnologyEuropeMabTheraOncologyPharmaceuticalPricingRare diseasesRegulationRoche

Deal between Ensemble and Alexion will target ultra-rare disorders


USA-based drug discovery firm Ensemble Therapeutics has announced the initiation of a drug discovery…

Alexion PharmaceuticalsBiotechnologyEnsemble TherapeuticsNorth AmericaPharmaceuticalRare diseasesResearch

Orphan status for Alexion's Solaris


US drugmaker Alexion Pharmaceuticals (Nasdaq: ALXN) says that Soliris (eculizumab), the company's first-in-class…

Alexion PharmaceuticalsBiotechnologyEuropeNeurologicalPharmaceuticalRare diseasesRegulationSolaris

Lupus therapeutics boast blockbuster hopefuls as R&D continues, says GlobalData


Lupus therapeutics are lacking, and patients with moderate forms of the disease are greatly underserved,…

BenlystaBiotechnologyBristol-Myers SquibbepratuzumabGlaxoSmithKlineImmunologicalsMarkets & MarketingOrenciaPharmaceuticalRare diseasesResearchUCB

European Medicines Agency simplifies processing of fee reductions for orphan medicines


The European Medicines Agency has cancelled the procedure by which sponsors of medicines with an orphan…

BiotechnologyEuropeFinancialPharmaceuticalRare diseasesRegulation

MoCA report a welcome stepping stone, but access to orphan medicines remains unfinished business, say trade groups


The three leading pharma/biotech trade bodies, EuropaBio, the European Federation of Pharmaceutical Industries…

BiotechnologyEuropeHealthcarePharmaceuticalPoliticsRare diseasesRegulation

Acceleron and Shire not to restart ACE-031 program


Privately-held US biotech firm Acceleron Pharma says that Ireland-headquartered Shire (LSE: SHP) has…

Acceleron PharmaACE-031BiotechnologyLicensingPharmaceuticalRare diseasesResearchShire

Orphan diseases' appeal lies in return on investment, says Evaluate


For many years, Big Pharma's model of success has been the blockbuster: drugs that sell more than $1…

BiotechnologyFinancialMarkets & MarketingPharmaceuticalRare diseasesResearch

Merck Serono's EPP funds EspeRare Foundation, which will reposition rimeporide


Merck Serono, the biotech unit of Germany's Merck KGaA (MRK: DE) has announced the launch of the EspeRare…

BiotechnologyEspeRare FoundationFinancialMerck KGaAMerck SeronoPharmaceuticalRare diseasesResearchrimeporide

Pfizer and Protalix' Gaucher drug Uplyso approved in Brazil


US drugs behemoth Pfizer (NYSE: PFE) and Israel-based Protalix BioTherapeutics (NYSE MKT:PLX) said that…

BiotechnologyElelysoPfizerPharmaceuticalProtalix BioTherapeuticsRare diseasesRegulationSouth AmericaUplyso

Shire acquires Premacure to expand in new therapy area


In a second acquisition so far this year, Ireland-headquartered drugmaker Shire (LSE: SHP) said on Tuesday…

BiotechnologyMergers & AcquisitionsOphthalmicsPharmaceuticalPremacureRare diseasesShire

Rare diseases research gets 144 million-euro funding from European Commission


On Rare Disease Day 2013 (February 28), the European Commission announced 144 million euros ($189.5 million)…

BiotechnologyEuropeFinancialPharmaceuticalRare diseasesResearch

Global orphan drugs market led by Roche's rituximab


The global orphan drugs market is estimated to have increased at a compound annual growth rate (CAGR)…

BiotechnologyLucentisMarkets & MarketingNovartisPharmaceuticalRare diseasesRituxanRoche

Genzyme's eliglustat meets endpoints in Ph III studies for Gaucher disease


US biotech firm Genzyme, a subsidiary if French drug major Sanofi (Euronext: SAN) has released positive…

BiotechnologyeliglustatGenzymePharmaceuticalRare diseasesResearchSanofi

EMA Committee for Orphan Medicinal Products - strengthened interactions with patients and international partners


"The European Medicines Agency's Committee for Orphan Medicinal Products (COMP) is stepping into another…

BiotechnologyEuropePharmaceuticalRare diseasesRegulation

Regulatory news for Hyperion's Ravicti and Seattle Genetics' Adcetris


The US Food and Drug Administration on Friday approved Hyperion Therapeutics' (Nasdaq: HPTX) Ravicti…

AdcetrisBiotechnologyHyperion TherapeuticsNorth AmericaOncologyPatentsPharmaceuticalRare diseasesRavictiRegulationSeattle Genetics

FDA clears Sanofi and Isis' Kynamro for inherited cholesterol disorder


The US Food and Drug Administration yesterday approved Sanofi (Euronext: SAN) subsidiary Genzyme's orphan…

BiotechnologyCardio-vascularGenzymeIsis PharmaceuticalsKynamroNorth AmericaPharmaceuticalRare diseasesRegulationSanofi

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