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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

FDA expands approval of Genzyme’s Lumizyme to treat Pompe disease

FDA expands approval of Genzyme’s Lumizyme to treat Pompe disease

02-08-2014

The US Food and Drug Administration on Friday approved Lumizyme (alglucosidase alfa) for treatment of…

BiotechnologyGenzymeLumizymeMyozymeRare diseasesRegulationSanofiUSA

Orphan status in Europe for Alexion’s Soliris in myasthenia gravis

Orphan status in Europe for Alexion’s Soliris in myasthenia gravis

01-08-2014

The European Commission has granted orphan drug designation (ODD) to US drugmaker Alexion Pharmaceuticals’…

Alexion PharmaceuticalsBiotechnologyEuropeEuropean CommissionMyasthenia gravisNeurologic disorderRare diseasesRegulationSoliris

US breakthrough status for InterMune’s pirfenidone

US breakthrough status for InterMune’s pirfenidone

21-07-2014

US biotech firm InterMune’s Esbriet (pirfenidone) has been granted Breakthrough Therapy designation…

BiotechnologyEsbrietFood and Drug AdministrationInterMunepirfenidoneRare diseasesRegulationRespiratory and PulmonaryUSA

Pharming and Salix gain US approval for Ruconest

Pharming and Salix gain US approval for Ruconest

17-07-2014

Netherlands-based Pharming Group and Salix Pharmaceuticals have received approval from the US Food and…

BiotechnologyPharmingRare diseasesRegulationRuconestSalix PharmaceuticalsUSA

Sarepta tanks despite positive data on eteplirsen in Duchenne MD

Sarepta tanks despite positive data on eteplirsen in Duchenne MD

11-07-2014

US RNA-based drug developer Sarepta Therapeutics saw its shares plunge, despite the company releasing…

BiotechnologyeteplirsenRare diseasesRegulationResearchSarepta TherapeuticsUSA

Baxter buys AesRx, gaining rights to investigational SCD drug

Baxter buys AesRx, gaining rights to investigational SCD drug

10-07-2014

US drugmaker Baxter International said yesterday that it has acquired AesRx, a private US biopharmaceutical…

Aes-103AesRxBaxter InternationalBiotechnologyHematologyMergers & AcquisitionsRare diseasesUSA

US orphan status for Mirati’s mocetinostat and Alexion’s Soliris

US orphan status for Mirati’s mocetinostat and Alexion’s Soliris

17-06-2014

USA-based Mirati Therapeutics has been granted Orphan Drug designation by the US Food and Drug Administration…

BiotechnologyOncologyRare diseasesRegulation

uniQure signs up Medison to distribute Glybera in Israel

uniQure signs up Medison to distribute Glybera in Israel

29-05-2014

Netherlands-based human gene therapy firm uniQure has entered an exclusive distribution agreement with…

BiotechnologyGlyberaLicensingMedison PharmaRare diseasesRest of the WorlduniQure

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

Amicus Thera leaps on positive migalastat results

29-04-2014

US biotech firm Amicus Therapeutics (has released positive 12- and 24-month data from its first Phase…

Amicus TherapeuticsBiotechnologymigalastatRare diseasesResearch

US FDA clears pathway for Sarepta’s eteplirsen filing by year end

22-04-2014

US RNA-based drug developer Sarepta Therapeutics it plans to submit a New Drug Application to the US…

BiotechnologyeteplirsenNorth AmericaRare diseasesRegulationSarepta TherapeuticsUSA

SOBI assumes direct responsibility for rare disease drug Orfadin in the USA and Canada

SOBI assumes direct responsibility for rare disease drug Orfadin in the USA and Canada

01-04-2014

Biotech firm Swedish Orphan Biovitrum has assumed direct responsibility for the distribution of its proprietary…

BiotechnologyMarkets & MarketingNorthern EuropeOrfadinRare diseasesSobi

Promising mid-stage results for Prosensa’s drisapersen in DMD boys

19-03-2014

Shares of Dutch biotech firm Prosensa Holding rose 3.85% to $7.02 after the firm reported encouraging…

BiotechnologydrisapersenGlaxoSmithKlineProsensaRare diseasesResearch

More positive Ph III data for Amgen’s evolocumab

More positive Ph III data for Amgen’s evolocumab

17-03-2014

Leading independent biotech firm Amgen has released more positive clinical trial data with its cholesterol…

AmgenBiotechnologyCardio-vascularevolocumabRare diseasesResearch

Six-year follow-up data from Glybera treated patients

Six-year follow-up data from Glybera treated patients

17-03-2014

Netherlands-based human gene company uniQure and privately-held Italian drugmaker Chiesi Farmaceutici…

BiotechnologyChiesi FarmaceuticiGlyberaRare diseasesResearchuniQure

Marina Biotech to use its therapeutics platform to develop drugs for rare diseases

11-03-2014

US RNAi-based drug discover Marina Biotech says that it plans to broadly apply its RNAi, antisense and…

BiotechnologyFinancialMarina BiotechRare diseasesResearch

UK’s BIA finds government unwilling to set price cap for orphan disease treatment

UK’s BIA finds government unwilling to set price cap for orphan disease treatment

11-03-2014

The majority of Members of Parliament in the UK believe there should not be a maximum price per patient…

BiotechnologyPoliticsPricingRare diseasesUK

Hawaii Biotech awarded $5.5million government grant for botulism therapy

Hawaii Biotech awarded $5.5million government grant for botulism therapy

05-03-2014

Privately-held US biotech company Hawaii Biotech has been awarded a contract to continue developing drugs…

BiotechnologyFinancialHawaii BiotechRare diseasesResearchUSA

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