Biotechnology - Rare diseases, North America


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FDA backs Kalydeco for eight additional mutations that cause cystic fibrosis


The US Food and Drug Administration approved biotech firm Vertex Pharmaceuticals’ supplemental New…

BiotechnologyKalydecoNorth AmericaRare diseasesRegulationUSAVertex

FDA approves Northera for neurogenic orthostatic hypotension

FDA approves Northera for neurogenic orthostatic hypotension


Shares of US biotech firm Chelsea Therapeutics soared 38% to $6.75 in after-hours trading yesterday,…

BiotechnologyChelsea TherapeuticsDainippon Sumitomo PharmaNorth AmericaNortheraRare diseasesRegulationUSA

Sarepta plunges as FDA questions eteplirsen trial results

Sarepta plunges as FDA questions eteplirsen trial results


Shares of US RNA-based drug developer Sarepta Therapeutics plummeted in pre-market trading on November…

BiotechnologydrisaperseneteplirsenGlaxoSmithKlineNorth AmericaRare diseasesRegulationResearchSarepta Therapeutics

Genzyme to invest $80 million to expand capacity for Fabrazyme

Genzyme to invest $80 million to expand capacity for Fabrazyme


French drug major Sanofi’s US biotech subsidiary Genzyme says that it is investing $80 million to build…

BiotechnologyFabrazymeFinancialGenzymeNorth AmericaProductionRare diseasesSanofi

Sarepta to file NDA for its Duchenne muscular dystrophy compound


US RNA-based drug developer Sarepta Therapeutics (Nasdaq: SRPT) plans to submit a New Drug Application…

BiotechnologyeteplirsenNorth AmericaRare diseasesRegulationSarepta Therapeutics

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

US FDA accepts Ruconest filing from Santarus and Pharming


US drugmaker Santarus (Nasdaq: SNTS) and Netherlands-based Pharming Group (NYSE Euronext: PHARM) say…

BiotechnologyCardio-vascularNorth AmericaPharmingRare diseasesRegulationRuconestSantarus

BioMarin files BLA for Vimizim in USA


US drugmaker BioMarin Pharmaceutical (Nasdaq: BMRN) has submitted a Biologics License Application (BLA)…

BioMarin PharmaceuticalBiotechnologyNorth AmericaRare diseasesRegulationVimizim

FTC files Amicus Brief, saying improper use of restricted drug distribution programs may impede generic competition


The US Federal Trade Commission has filed an amicus brief in the US District Court for the District of…

ActavisActelionApotexBiotechnologyGenericsLegalNorth AmericaRare diseasesRespiratory and PulmonaryRoxaneTracleerZavesca

Biogen Idec files for US approval of long-acting hemophilia A treatment


US biotech firm Biogen Idec (Nasdaq: BIIB) says it has submitted a Biologics License Application (BLA)…

Biogen IdecBiotechnologyNorth AmericaRare diseasesRegulationrFVIIIFc

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

FDA approves Aegerion's new orphan drug Juxtapid for rare cholesterol disorder


The US Food and Drug Administration has recently approved emerging US biotech firm Aegerion Pharmaceuticals'…

Aegerion PharmaceuticalsBiotechnologyCardio-vascularJuxtapidlomitapideNorth AmericaRare diseasesRegulation

Isis Pharma/GlaxoSmithKline's ISIS-TTR Rx gains US fast-track designation


In what has been a busy newsflow few days for USA-based Isis Pharmaceutical (Nasdaq: ISIS), the company…

BiotechnologyGlaxoSmithKlineIsis PharmaceuticalsISIS-TTR.RXLicensingNorth AmericaPharmaceuticalRare diseasesRegulation

Health Canada approves Vertex' Kalydeco; and Sanofi Pasteur's Menactra


Vertex Pharmaceuticals (Nasdaq: VRTX) says that Health Canada has approved Kalydeco (ivacaftor), the…

BiotechnologyKalydecoMenactraNorth AmericaPharmaceuticalRare diseasesRegulationSanofiSanofi PasteurVaccinesVertex

FDA advisory panel votes 9 to 6 to approve Genzyme's Kynamro


The US Food and Drug Administration's Endocrinologic and Metabolic Drugs Advisory yesterday voted by…

BiotechnologyCardio-vascularGenzymeIsis PharmaceuticalsKynamroNorth AmericaPharmaceuticalRare diseasesRegulationSanofi

New Canadian rare disease strategy welcomed as offering hope and solutions for patients


The Canadian government this week announced a new national framework to improve access to "orphan drugs"…

BiotechnologyNorth AmericaPharmaceuticalRare diseasesRegulationResearch

Positive Ph III results for Biogen Idec and SOBI's hemophilia B agent


US biotech firm Biogen Idec (Nasdaq: BIIB) and partner Swedish Orphan Biovitrum (STO: SOBI) have announced…

Biogen IdecBiotechnologyEuropeNorth AmericaPharmaceuticalRare diseasesRegulationResearchrFIXFcSobiSwedish Orphan Biovitrum

Auxilium and Actelion get Xiaflex approval in Canada


US company Auxilium Pharmaceuticals (Nasdaq: AUXL) and the Canadian subsidiary of Actelion (SIX: ATLN),…

ActelionAuxilium PharmaceuticalsBiotechnologyNorth AmericaRare diseasesRegulationXiaflex

Chelsea Thera gets disappointing news from FDA on Northera


US biotech firm Chelsea Therapeutics International (Nasdaq: CHTP) shares tumbled in pre-market trading…

BiotechnologyCardio-vascularChelsea TherapeuticsNorth AmericaNortheraRare diseasesRegulation

Aegerion files for lomitapide approval in USA and Europe


Emerging US biotech firm Aegerion Pharmaceuticals (Nasdaq: AEGR) has submitted a New Drug Application…

Aegerion PharmaceuticalsBiotechnologyEuropelomitapideNorth AmericaRare diseasesRegulation

EMA approves Shire Vpriv plant in USA


Ireland-headquartered biopharma firm Shire (LSE: SHP) says that the European Medicines Agency's Committee…

BiotechnologyFinancialNorth AmericaPharmaceuticalRare diseasesRegulationReplagalShireVpriv

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