Pharmaceutical - Pharmaceutical, North America, Rare diseases

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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 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

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

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

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

FDA grants "Breakthrough Therapy" status for Alexion's asfotase in HPP; issues CRL for Valeant's efinaconazole

28-05-2013

US drugmaker Alexion Pharmaceuticals (Nasdaq: ALXN) says that the Food and Drug Administration has granted…

Alexion PharmaceuticalsAntibiotics and Infectious diseasesasfotase alfaefinaconazoleNorth AmericaPharmaceuticalRare diseasesRegulationValeant Pharmaceuticals

Novartis' Ilaris gets new indication of childhood arthritis from US FDA

10-05-2013

Swiss pharmaceutical major Novartis (NIVN: VX) said this morning (May 10) that the US Food and Drug Administration…

Anti-Arthritics/RheumaticsNorth AmericaPharmaceuticalRare diseasesRegulation

Cornerstone gains US rights to Pertzye for EPI due to cystic fibrosis patients

09-05-2013

US specialty pharma company Cornerstone Therapeutics (Nasdaq: CRTX) has entered into an agreement with…

Cornerstone TherapeuticsDigestive CareLicensingNorth AmericaPertzyePharmaceuticalRare diseases

FDA approves Raptor's Procysbi for rare genetic condition; and Plan B One-Step

01-05-2013

US drugmaker Raptor Pharmaceuticals (Nasdaq: RPTP) saw its share price rise 7.5% to $7.06 in mid-afternoon…

North AmericaPharmaceuticalPlan B One-StepProcysbiRaptor PharmaceuticalsRare diseasesRegulationReproductiveTeva Pharmaceutical IndustriesWomen's Health

Pharming and Santarus file BLA for Ruconest with US FDA

18-04-2013

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

North AmericaPharmaceuticalPharmingRare diseasesRegulationRuconestSantarus

US FDA accepts NDAs for Otsuka's tolvaptan and BTG's Varisolve

12-04-2013

Japanese mid-sized drugmaker Otsuka Pharmaceutical (TYO: 4578) says that the US Food and Drug Administration…

BTGCardio-vascularNephrology and HepatologyNorth AmericaOtsukaPharmaceuticalRare diseasesRegulationtolvaptanVarisolve

US FDA calls for new trial with Pharmaxis' CF drug Bronchitol

19-03-2013

Australian pharma company Pharmaxis (ASX: PXS) says it has received a complete response letter from the…

BronchitolNorth AmericaPharmaceuticalPharmaxisRare diseasesRegulationRespiratory and Pulmonary

CSL Behring gains expanded use for Corifact in USA

06-03-2013

Corifact, Factor XIII Concentrate (Human), to include the peri-operative management of surgical bleeding…

CorifactCSL BehringCSL LimitedNorth AmericaPharmaceuticalRare diseasesRegulation

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