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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

EMA urging firms with Ebola candidates to seek orphan status

21-10-2014

The European Medicines Agency is aiming to encourage developers of treatments or vaccines against Ebola…

Anti-viralsEuropePharmaceuticalRare diseasesRegulationResearchUSA

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

NICE draft guidance recommends Alexion’s Soliris for very rare life-threatening blood disorder

NICE draft guidance recommends Alexion’s Soliris for very rare life-threatening blood disorder

04-09-2014

A drug to treat a very rare blood disorder affecting around 200 people in England has been recommended…

Alexion PharmaceuticalsBiotechnologyHematologyPricingRare diseasesRegulationSolirisUK

FDA approves Elelyso for pediatric use in Gaucher disease

FDA approves Elelyso for pediatric use in Gaucher disease

29-08-2014

The US Food and Drug Administration has approved US pharma giant Pfizer’s Elelyso (taliglucerase alfa)…

ElelysoPfizerPharmaceuticalProtalix BioTherapeuticsRare diseasesRegulationUSA

FDA backs added indication for GSK’s Promacta

FDA backs added indication for GSK’s Promacta

27-08-2014

The Food and Drug Administration has approved UK pharma giant GlaxoSmithKline’s supplemental New Drug…

GlaxoSmithKlineHematologyLigand PharmaceuticalsPharmaceuticalPromactaRare diseasesRegulationRevoladeUSA

Positive Ph III data for Amicus’ migalastat as Fabry monotherapy

Positive Ph III data for Amicus’ migalastat as Fabry monotherapy

21-08-2014

US biopharma company Amicus Therapeutics has revealed positive 18-month data from its second Phase III…

Amicus TherapeuticsBiotechnologymigalastatRare diseasesRegulationResearch

IQWiG finds added benefit for Novartis’ Jakavi in myelofibrosis

IQWiG finds added benefit for Novartis’ Jakavi in myelofibrosis

20-08-2014

In an early benefit assessment under the German Act on the Reform of the Market for Medicinal Products,…

Federal Joint CommitteeGermanyJakaviMyelofibrosisNovartisOncologyPharmaceuticalPricingRare diseasesRegulation

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

Patient involvement pilot for orphan drugs launches in Canada

Patient involvement pilot for orphan drugs launches in Canada

07-08-2014

Canada’s Minister of Health, Rona Ambrose, says that the government has launched a pilot project targeting…

CanadaHealth CanadaPharmaceuticalRare diseasesRegulationResearchRocheTreatment of chronic lymphocytic leukemia

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

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

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

Kyowa Hakko Kirin files for KW-3357 approval in Japan

Kyowa Hakko Kirin files for KW-3357 approval in Japan

01-08-2014

Japanese drugmaker Kyowa Hakko Kirin has filed an application for marketing approval with the Ministry…

HematologyJapanKW-3357Kyowa Hakko KirinPharmaceuticalRare diseasesRegulationThrombus

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

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

EU orphan designations for OPKO's clotting Factor VIIa-CTP

EU orphan designations for OPKO's clotting Factor VIIa-CTP

10-07-2014

The European Committee for Orphan Medicinal Products (COMP) gave a positive opinion recommending the…

EuropeFactor VIIa-CTPHematologyOPKO HealthPharmaceuticalRare diseasesRegulation

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

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