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Prosensa and CureDuchenne expand DMD collaboration with $7 million funding boost

Prosensa and CureDuchenne expand DMD collaboration with $7 million funding boost

12-08-2014

Dutch biotech company Prosensa will receive a 5 million euros ($7 million) funding boost from US non-profit…

Duchenne Muscular DystrophyFinancialNetherlandsPharmaceuticalProsensaRare diseasesResearchUSA

US and European orphan status for AbbVie’s glioblastoma candidate

US and European orphan status for AbbVie’s glioblastoma candidate

04-08-2014

Both the European Medicines Agency and the US Food and Drug Administration have granted orphan drug designation…

AbbVieABT-414Brain tumorEuropeGlioblastoma multiformeMarkets & MarketingOncologyPharmaceuticalRare diseasesRegulationUS Food and Drug AdministrationUSA

Shire finally accepts AbbVie’s takeover offer

Shire finally accepts AbbVie’s takeover offer

18-07-2014

After a protracted and at times hostile courtship, Ireland-headquartered drugmaker Shire has finally…

AbbVieHumiraMergers & AcquisitionsNeurologicalPharmaceuticalRare diseasesShireUSA

Shire confirms it has entered into talks with AbbVie

Shire confirms it has entered into talks with AbbVie

12-07-2014

Following reports by the Bloomberg news service, on Friday Ireland-based drugmaker Shire confirmed that…

AbbVieIrelandMergers & AcquisitionsNeurologicalPharmaceuticalRare diseasesShireUSA

Orphan drug approvals rise, but high costs pose challenges for patients

Orphan drug approvals rise, but high costs pose challenges for patients

10-07-2014

Although the pace of approvals for new orphan drugs - medicines that treat relatively rare conditions…

EuropePharmaceuticalPricingRare diseasesRegulationUSA

Novo Nordisk gains FDA backing for new NovoSeven RT indication

Novo Nordisk gains FDA backing for new NovoSeven RT indication

09-07-2014

Danish diabetes care giant Novo Nordisk’s hemophilia treatment NovoSeven RT (coagulation factor VIIa…

HematologyNovo NordiskNovoSevenNovoSeven RTPharmaceuticalRare diseasesRegulationUSA

FDA orphan status for Insys’ cannabidiol as treatment for Dravet syndrome

FDA orphan status for Insys’ cannabidiol as treatment for Dravet syndrome

03-07-2014

US specialty pharma firm Insys Therapeutics yesterday revealed that the US Food and Drug Administration…

cannabidiolInsys TherapeuticsNeurologicalPharmaceuticalRare diseasesRegulationUSA

US orphan status for Insys Therapeutics’ cannabidiol for rare form of epilepsy

US orphan status for Insys Therapeutics’ cannabidiol for rare form of epilepsy

25-06-2014

US specialty pharma firm Insys Therapeutics says that the US Food and Drug Administration has granted…

cannabidiolInsys TherapeuticsNeurologicalPharmaceuticalRare diseasesRegulationUSA

UK’s Summit sets up operations in the USA

UK’s Summit sets up operations in the USA

17-06-2014

UK drug discovery firm Summit, which is focused on therapies for Duchenne muscular dystrophy and Clostridium…

Antibiotics and Infectious diseasesBoardroomManagementPharmaceuticalRare diseasesResearchSummit PharmaceuticalsUSA

US orphan status for EffRx’ EX404 for pediatric polycystic ovary syndrome

US orphan status for EffRx’ EX404 for pediatric polycystic ovary syndrome

11-06-2014

Swiss drug delivery specialist EffRx Pharmaceuticals says the US Food and Drug Administration has granted…

EffRx PharmaceuticalsEX404metforminPharmaceuticalRare diseasesRegulationUSAWomen's Health

US FDA grants Fast Track designation for GW Pharma’s Epidiolex in Dravet syndrome

US FDA grants Fast Track designation for GW Pharma’s Epidiolex in Dravet syndrome

06-06-2014

The US Food and Drug Administration has granted Fast Track designation to UK-based GW Pharmaceuticals’…

epidiolexGW PharmaceuticalsPharmaceuticalRare diseasesRegulationTreatment of Dravet syndromeUSA

Retrophin acquires US Thiola rights from Mission Pharmacal

Retrophin acquires US Thiola rights from Mission Pharmacal

30-05-2014

US drugmaker Retrophin has entered into a license agreement with Mission Pharmacal, a private company…

LicensingMission PharmacalNephrology and HepatologyPharmaceuticalRare diseasesRetrophinThiolaUSA

Ipsen takes step forward in the resupply of Increlex for the USA

Ipsen takes step forward in the resupply of Increlex for the USA

13-05-2014

Following previous availability problems, French drugmaker Ipsen said today that a supply of Increlex…

IncrelexIpsenMarkets & MarketingPharmaceuticalProductionRare diseasesUSA

FDA backs Janssen’s Sylvant for rare disease

24-04-2014

The US Food and Drug Administration yesterday approved Johnson & Johnson subsidiary Janssen Biotech’s…

Janssen BiotechJohnson & JohnsonNorth AmericaOncologyPharmaceuticalRare diseasesRegulationsiltuximabSylvantUSA

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

Sanofi renews alliance with Regulus and ups investment by $10 million

Sanofi renews alliance with Regulus and ups investment by $10 million

05-02-2014

US pharma company Regulus Therapeutics has renewed its strategic alliance with French drug major Sanofi…

FinancialFranceOncologyPharmaceuticalRare diseasesRegulus TherapeuticsResearchSanofiUSA

US FDA grants Edison’s EPI-743 orphan status for Friedreich's ataxia

US FDA grants Edison’s EPI-743 orphan status for Friedreich's ataxia

04-02-2014

Privately-held US pharma company Edison Pharmaceuticals has announced that the US Food and Drug Administration…

Edison PharmaceuticalsEPI-743PharmaceuticalRare diseasesRegulationUSA

Aegerion receives US subpoena over USA sales of Juxtapid

Aegerion receives US subpoena over USA sales of Juxtapid

13-01-2014

US biopharma company Aegerion Pharmaceuticals has received a subpoena from the US Department of Justice…

Aegerion PharmaceuticalsFinancialJuxtapidLegalPharmaceuticalRare diseasesUSA

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