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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

Extended US market exclusivity for BioMarin's rare disease drug Kuvan

14-04-2014

US drugmaker BioMarin Pharmaceutical says that the Food and Drug Administration has granted Kuvan (sapropterin…

BioMarin PharmaceuticalKuvanNorth AmericaPharmaceuticalRare diseasesRegulationUSA

US orphan designation for OPKO's longer-acting clotting Factor VIIa-CTP

US orphan designation for OPKO's longer-acting clotting Factor VIIa-CTP

03-03-2014

The US Food and Drug Administration has granted orphan drug designation to US drugmaker OPKO Health’s…

HematologyNorth AmericaOPKO HealthPharmaceuticalRare diseasesUSA

GSK files for US approval for Promacta for severe aplastic anemia

GSK files for US approval for Promacta for severe aplastic anemia

02-03-2014

UK pharma giant GlaxoSmithKline has submitted a supplemental New Drug Application to the US Food and…

GlaxoSmithKlineLigand PharmaceuticalsNorth AmericaOncologyPharmaceuticalPromactaRare diseasesRegulationUSA

FDA approves Myalept, the first drug to treat a rare metabolic disease

FDA approves Myalept, the first drug to treat a rare metabolic disease

26-02-2014

The US Food and Drug Administration has approved Myalept (metreleptin for injection) as replacement therapy…

AstraZenecaBristol-Myers SquibbmetreleptinMyaleptNorth AmericaPharmaceuticalRare diseasesRegulationUSA

US orphan drug market outlook 2018: Report

25-02-2014

In the largest market for orphan drugs, the USA, there was a shortage of adequate therapies for treating…

Markets & MarketingNorth AmericaPharmaceuticalRare diseasesRegulationUSA

FDA backs Kalydeco for eight additional mutations that cause cystic fibrosis

22-02-2014

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

19-02-2014

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

BiotechnologyChelsea TherapeuticsDainippon Sumitomo PharmaNorth AmericaNortheraRare diseasesRegulationUSA

FDA approves Vimizim to treat rare congenital enzyme disorder

FDA approves Vimizim to treat rare congenital enzyme disorder

17-02-2014

The US Food and Drug Administration on Friday (February 14) approved BioMarin Pharmaceutical’s Vimizim…

BioMarin PharmaceuticalNorth AmericaPharmaceuticalRare diseasesRegulationUSAVimizim

FDA approves Novo Nordisk’s Tretten to treat rare genetic clotting disorder

FDA approves Novo Nordisk’s Tretten to treat rare genetic clotting disorder

24-12-2013

The US Food and Drug Administration yesterday (December 23) approved Tretten, coagulation factor XIII…

HematologyNorth AmericaNovo NordiskNovoThirteenPharmaceuticalRare diseasesRegulationTretten

Mixed FDA advisory panel votes on Bristol-Myers and AstraZeneca's metreleptin

Mixed FDA advisory panel votes on Bristol-Myers and AstraZeneca's metreleptin

12-12-2013

In an 11 to one vote yesterday, the US Food and Drug Administration’s Endocrinologic and Metabolic…

AstraZenecaBristol-Myers SquibbMetabolicsmetreleptinNorth AmericaPharmaceuticalRare diseasesRegulation

US FDA accepts Nova Lab’s filing for mercaptopurine in childhood ALL

US FDA accepts Nova Lab’s filing for mercaptopurine in childhood ALL

04-12-2013

Privately-held UK drugmaker Nova Laboratories says the US Food and Drug Administration has accepted a…

MercaptopurineNorth AmericaNova LaboratoriesPharmaceuticalRare Disease TherapeuticsRare diseasesRegulationXaluprine

FDA committee recommends approval for BioMarin's Vimizim

FDA committee recommends approval for BioMarin's Vimizim

20-11-2013

A US Food and Drug Administration panel has voted in favor of approval of Vimizim for the treatment of…

BioMarin PharmaceuticalNorth AmericaPharmaceuticalRare diseasesRegulationUSAVimizim

GW Pharma receives FDA orphan drug designation for cannaboid-based agent

GW Pharma receives FDA orphan drug designation for cannaboid-based agent

15-11-2013

GW Pharmaceuticals (Nasdaq: GWPH) has been granted orphan drug designation by the US Food and Drug Administration…

EuropeGW PharmaceuticalsNorth AmericaPharmaceuticalRare diseasesRegulationUKUSA

Sarepta plunges as FDA questions eteplirsen trial results

Sarepta plunges as FDA questions eteplirsen trial results

12-11-2013

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

15-10-2013

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

BiotechnologyFabrazymeFinancialGenzymeNorth AmericaProductionRare diseasesSanofi

PhRMA calls for regulatory environment to support rare disease research

PhRMA calls for regulatory environment to support rare disease research

08-10-2013

US biopharmaceutical research companies are developing 452 new medicines for rare diseases, according…

North AmericaPharmaceuticalRare diseasesRegulationResearch

Janssen files for simultaneous US and EU approval for Castleman disease drug

04-09-2013

Janssen Research & Development, a subsidiary of US health care giant Johnson & Johnson (NYSE: JNJ), yesterday…

EuropeJanssenJohnson & JohnsonNorth AmericaOncologyPharmaceuticalRare diseasesRegulationsiltuximab

Sarepta to file NDA for its Duchenne muscular dystrophy compound

25-07-2013

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

18-07-2013

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

Alexion PharmaceuticalsBiotechnologyEnsemble TherapeuticsNorth AmericaPharmaceuticalRare diseasesResearch

FDA backs Baxter's Rixubis for hemophilia B; Breakthrough status for GSK's drisapersen

28-06-2013

The US Food and Drug Administration yesterday (June 27) approved Baxter International's (NYSE: BAX) Rixubis…

Baxter InternationaldrisapersenGlaxoSmithKlineNorth AmericaPharmaceuticalRare diseasesRegulationRixubis

US FDA accepts Ruconest filing from Santarus and Pharming

18-06-2013

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

BiotechnologyCardio-vascularNorth AmericaPharmingRare diseasesRegulationRuconestSantarus

BioMarin's Vimizim BLA accepted by FDA and MAA validated by EMA

30-05-2013

US drugmaker BioMarin Pharmaceutical (Nasdaq: BMRN) says that the Food and Drug Administration has accepted…

BioMarin PharmaceuticalEuropeNorth AmericaPharmaceuticalRare diseasesRegulationVimizim

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