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US FDA approves NPS Pharma’s Natpara

US FDA approves NPS Pharma’s Natpara

24-01-2015

The US Food and Drug Administration on Friday approved NPS Pharmaceuticals’ Natpara (parathyroid hormone)…

BiotechnologyNatparaNPS PharmaceuticalsRare diseasesRegulationShireUSA

Roche continues spending spree, with acquisition of French biotech firm Trophos

Roche continues spending spree, with acquisition of French biotech firm Trophos

16-01-2015

Continuing its recent spate of deal-making, Swiss pharma giant Roche has agreed to acquire Trophos, a…

BiotechnologyMergers & AcquisitionsolesoximeRare diseasesRocheSwitzerlandTrophos

Aegerion Pharma under SEC investigation

Aegerion Pharma under SEC investigation

14-01-2015

US biotech firm Aegerion Pharmaceuticals has revealed it is the subject of a US Securities and Exchange…

Aegerion PharmaceuticalsBiotechnologyBrazilCardio-vascularJuxtapidLegalMarkets & MarketingRare diseasesUSA

Shire to spend $5.2 billion to buy NPS Pharma

Shire to spend $5.2 billion to buy NPS Pharma

12-01-2015

Recently jilted Ireland-headquartered Shire says it is to acquire NPS Pharmaceuticals, confirming recent…

BiotechnologyEuropeGattexMergers & AcquisitionsNatparaNPS PharmaceuticalsRare diseasesRevestiveShire

US FDA approves wider use for Vertex’ Kalydeco

US FDA approves wider use for Vertex’ Kalydeco

30-12-2014

US biotech firm Vertex Pharmaceuticals says the US Food and Drug Administration has approved a supplemental…

BiotechnologyKalydecoRare diseasesRegulationUSAVertex Pharmaceuticals

BioMarin’s Vimizim approved in Japan for Morquio A syndrome

BioMarin’s Vimizim approved in Japan for Morquio A syndrome

29-12-2014

US biotech firm BioMarin Pharmaceutical has received approval from Japan’s Ministry of Health, Labor…

BioMarin PharmaceuticalBiotechnologyJapanRare diseasesRegulationVimizim

FDA approves new indication for Amgen's Xgeva

FDA approves new indication for Amgen's Xgeva

08-12-2014

USA-based Amgen says the US Food and Drug Administration has approved a new indication for Xgeva (denosumab),…

AmgenBiotechnologyOncologyRare diseasesRegulationUSAXgeva

Credit Suisse sees PTC Therapeutics as a top stock pick

05-12-2014

Credit Suisse has released a biotech research report from analysts Jason Kantor and Jeremiah Shepard…

BiotechnologyFinancialNeurologicalPTC TherapeuticsRare diseasesRegulationTranslarna

NICE final draft guidance recommends Soliris, but saying it is “very expensive”

NICE final draft guidance recommends Soliris, but saying it is “very expensive”

27-11-2014

The UK National Institute for Health and Care Excellence (NICE) has today issued final draft guidance…

Alexion PharmaceuticalsBiotechnologyPricingRare diseasesRegulationSolirisUK

Royalty Pharma pays $3.3 billion for CFF royalty rights to Kalydeco

19-11-2014

Royalty Pharma says it has acquired royalties on Vertex Pharmaceuticals' cystic fibrosis treatments,…

BiotechnologyFinancialKalydecoRare diseasesRoyalty PharmaVertex Pharmaceuticals

Dicerna licenses rights to Tekmira technology

Dicerna licenses rights to Tekmira technology

18-11-2014

US RNAi therapeutics specialist Dicerna Pharmaceuticals has entered a licensing agreement for to use…

BiotechnologyDCR-PH1Dicerna PharmaceuticalsLicensingRare diseasesTekmira PharmaceuticalsUSA

Vertex seeks regulatory approval for lumacaftor/ivacaftor combination for CF

05-11-2014

US biotech firm Vertex Pharmaceuticals has filed for marketing approval for a fully co-formulated combination…

BiotechnologyEuropeivacaftorlumacaftorRare diseasesRegulationUSAVertex Pharmaceuticals

Pharming strengthens balance sheet with $20 million milestone from Salix

Pharming strengthens balance sheet with $20 million milestone from Salix

04-11-2014

Dutch biotech company Pharming Group says it has received a $20 million milestone payment from the USA’s…

BiotechnologyFinancialPharming GroupRare diseasesRuconestSalix Pharmaceuticals

FDA advisory backs expanded use of Vertex’ CF drug

22-10-2014

There was good news for US biotech firm Vertex Pharmaceuticals yesterday, as a US Food and Drug Administration…

BiotechnologyKalydecoRare diseasesRegulationUSAVertex Pharmaceuticals

Orphan status for Horizon Pharma’s Actimmune in Friedreich's ataxia

Orphan status for Horizon Pharma’s Actimmune in Friedreich's ataxia

04-10-2014

The US Food and Drug Administration has granted orphan drug designation for Ireland-headquartered specialty…

ActimmuneBiotechnologyHorizon PharmaRare diseasesRegulationUSA

Genzyme collaborates on gene therapy for rare disease that causes childhood blindness

Genzyme collaborates on gene therapy for rare disease that causes childhood blindness

25-09-2014

French pharma major Sanofi subsidiary Genzyme has entered into a research collaboration with the USA’s…

BiotechnologyFranceGenzymeOphthalmicsRare diseasesSanofi

Australian government announces funding available for Soliris

18-09-2014

The Pharmaceutical Benefits Advisory Committee (PBAC) has recommended to the Australian government that…

Alexion PharmaceuticalsAustraliaBiotechnologyFinancialPricingRare diseasesSoliris

NICE draft guidance recommends Alexion’s Soliris for very rare life-threatening blood disorder

NICE draft guidance recommends Alexion’s Soliris for very rare life-threatening blood disorder

04-09-2014

A drug to treat a very rare blood disorder affecting around 200 people in England has been recommended…

Alexion PharmaceuticalsBiotechnologyHematologyPricingRare diseasesRegulationSolirisUK

bluebird bio poised to advance gene therapy pipeline

bluebird bio poised to advance gene therapy pipeline

02-09-2014

US clinical-stage biotech firm bluebird bio is advancing a pipeline focused on gene therapy, a rapidly…

Biotechnologybluebird bioCelgene Corp.OncologyRare diseasesResearch

Positive Ph III data for Amicus’ migalastat as Fabry monotherapy

Positive Ph III data for Amicus’ migalastat as Fabry monotherapy

21-08-2014

US biopharma company Amicus Therapeutics has revealed positive 18-month data from its second Phase III…

Amicus TherapeuticsBiotechnologymigalastatRare diseasesRegulationResearch

Genzyme’s Cerdelga gets FDA approval for Gaucher disease

Genzyme’s Cerdelga gets FDA approval for Gaucher disease

20-08-2014

The US Food and Drug Administration late yesterday approved Cerdelga (eliglustat) for the long-term treatment…

ActelionBiotechnologyCerdelgaCerezymeGenzymeRare diseasesRegulationSanofiUSAZavesca

Oncothyreon acquires Alpine Biosciences

Oncothyreon acquires Alpine Biosciences

12-08-2014

US biotech firm Oncothyreon says that it has acquired Alpine Biosciences, of Seattle, Washington, a privately…

Alpine BiosciencesBiotechnologyBusiness FinanceImmunotherapyMedicineMergers & AcquisitionsOncologyOncothyreonRare diseasesUSA

Chiesi and uniQure delay Glybera launch to add data

Chiesi and uniQure delay Glybera launch to add data

04-08-2014

Netherlands-based venture capital backed human gene therapy firm uniQure and privately-held Italian drugmaker…

BiotechnologyChiesi FarmaceuticiEuropeGlyberaOrphan disease lipoprotein lipase deficiencyPricingRare diseasesRegulationuniQure

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