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Shire acquires Meritage Pharma, strengthening rare diseases pipeline

Shire acquires Meritage Pharma, strengthening rare diseases pipeline

24-02-2015

In a second acquisition this year, Ireland-headquartered Shire revealed today that it has taken control…

BiotechnologyGastro-intestinalsMergers & AcquisitionsMeritage PharmaRare diseasesShire

Sosei acquires Heptares Therapeutics for up to $400 million

Sosei acquires Heptares Therapeutics for up to $400 million

23-02-2015

Japanese drugmaker Sosei Group says it has acquired Heptares Therapeutics, a clinical-stage private UK-based…

BiotechnologyHeptares TherapeuticsMergers & AcquisitionsNeurologicalRare diseasesSosei

EMA Orphan Drug status for aTyr’s Resolaris in FSHD

18-02-2015

Privately-held US biotech firm aTyr Pharma says that the European Commission has granted Orphan Drug…

ATyr PharmaBiotechnologyEuropeRare diseasesRegulationResolaris

Australia to subsidize Genzyme’s Pompe disease drug Myozyme for young patients

Australia to subsidize Genzyme’s Pompe disease drug Myozyme for young patients

17-02-2015

Australia’s Minister for Health Sussan Ley said today that she has approved a life-saving drug that…

AustraliaBiotechnologyFinancialGenzymeHealthcareMyozymeRare diseasesSanofi

Positive Ph III clinical trial extension results for Genzyme’s Cerdelga

Positive Ph III clinical trial extension results for Genzyme’s Cerdelga

13-02-2015

French pharma major Sanofi subsidiary Genzyme has reported positive extension study data from its Phase…

BiotechnologyCerdelgaGenzymeRare diseasesResearchSanofi

Strong 2014 financials from Shire, bolstered by

Strong 2014 financials from Shire, bolstered by "break fee"

12-02-2015

Shares of Ireland-headquartered Shire gained 4.6% to £49.97, after the drugmaker reported pretax profit…

BiotechnologyFinancialRare diseasesShire

Alexion’s Soliris price to be reviewed by Canadian regulator

07-02-2015

Canada’s Patented Medicine Prices review Board (PMPRB) will hold a public hearing in the matter of…

Alexion PharmaceuticalsBiotechnologyCanadaPricingRare diseasesSoliris

New biotech start-up OptiKira created by BioMotiv

New biotech start-up OptiKira created by BioMotiv

02-02-2015

USA-based BioMotiv, a drug development accelerator associated with The Harrington Project, the University…

BioMotivBiotechnologyDiabetesMergers & AcquisitionsOptiKiraRare diseasesResearch

First UK NICE highly specialized technologies guidance recommends Soliris

First UK NICE highly specialized technologies guidance recommends Soliris

28-01-2015

England’s health costs watchdog the National Institute for Health and care Excellence has today issued…

Alexion PharmaceuticalsBiotechnologyHematologyPricingRare diseasesRegulationSolarisUK

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

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