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UPDATE: FDA approves Eisai’s Lenvima for a type of thyroid cancer and expands use of Banzel

UPDATE: FDA approves Eisai’s Lenvima for a type of thyroid cancer and expands use of Banzel

14-02-2015

The US Food and Drug Administration on Friday granted approval for Japanese pharma major Eisai’s Lenvima…

BanzelEisailenvatinibLenvimaNeurologicalOncologyPharmaceuticalRare diseasesRegulationUSA

GSK files for EU expanded indication for Revolade

GSK files for EU expanded indication for Revolade

06-02-2015

UK pharma major GlaxoSmithKline has filed a submission with the European Medicines Agency for a variation…

eltrombopagEuropeGlaxoSmithKlinePharmaceuticalRare diseasesRegulationRevolade

First UK NICE highly specialized technologies guidance recommends Soliris

First UK NICE highly specialized technologies guidance recommends Soliris

28-01-2015

England’s health costs watchdog the National Institute for Health and care Excellence has today issued…

Alexion PharmaceuticalsBiotechnologyHematologyPricingRare diseasesRegulationSolarisUK

US FDA approves NPS Pharma’s Natpara

US FDA approves NPS Pharma’s Natpara

24-01-2015

The US Food and Drug Administration on Friday approved NPS Pharmaceuticals’ Natpara (parathyroid hormone)…

BiotechnologyNatparaNPS PharmaceuticalsRare diseasesRegulationShireUSA

US FDA approves wider use for Vertex’ Kalydeco

US FDA approves wider use for Vertex’ Kalydeco

30-12-2014

US biotech firm Vertex Pharmaceuticals says the US Food and Drug Administration has approved a supplemental…

BiotechnologyKalydecoRare diseasesRegulationUSAVertex Pharmaceuticals

BioMarin’s Vimizim approved in Japan for Morquio A syndrome

BioMarin’s Vimizim approved in Japan for Morquio A syndrome

29-12-2014

US biotech firm BioMarin Pharmaceutical has received approval from Japan’s Ministry of Health, Labor…

BioMarin PharmaceuticalBiotechnologyJapanRare diseasesRegulationVimizim

FDA approves Teva’s Granix and Galderma’s Soolantra

FDA approves Teva’s Granix and Galderma’s Soolantra

24-12-2014

The Food and Drug Administration has approved Israeli generics giant Teva Pharmaceutical Industries’…

DermatologicalsGaldermaGranixIvermectinPharmaceuticalRare diseasesRegulationSoolantraTeva Pharmaceutcal IndustriesUSA

Novartis gains US FDA approval of Signifor for acromegaly

Novartis gains US FDA approval of Signifor for acromegaly

16-12-2014

The US Food and Drug Administration has approved Swiss pharma giant Novartis’ Signifor (pasireotide)…

NovartisPharmaceuticalRare diseasesRegulationSigniforUSA

FDA approves new indication for Amgen's Xgeva

FDA approves new indication for Amgen's Xgeva

08-12-2014

USA-based Amgen says the US Food and Drug Administration has approved a new indication for Xgeva (denosumab),…

AmgenBiotechnologyOncologyRare diseasesRegulationUSAXgeva

Takeda receives Breakthrough designation for ixazomib

Takeda receives Breakthrough designation for ixazomib

05-12-2014

Japanese drug major Takeda Pharmaceutical’s has been granted Breakthrough Therapy designation from…

ixazomibPharmaceuticalRare diseasesRegulationTakeda PharmaUSA

Credit Suisse sees PTC Therapeutics as a top stock pick

05-12-2014

Credit Suisse has released a biotech research report from analysts Jason Kantor and Jeremiah Shepard…

BiotechnologyFinancialNeurologicalPTC TherapeuticsRare diseasesRegulationTranslarna

NICE final draft guidance recommends Soliris, but saying it is “very expensive”

NICE final draft guidance recommends Soliris, but saying it is “very expensive”

27-11-2014

The UK National Institute for Health and Care Excellence (NICE) has today issued final draft guidance…

Alexion PharmaceuticalsBiotechnologyPricingRare diseasesRegulationSolirisUK

OPEN Act introduced in USA to repurpose drugs for rare diseases

OPEN Act introduced in USA to repurpose drugs for rare diseases

26-11-2014

Last week, US Representatives Gus Bilirakis (Republican, Florida) and G K Butterfield (Democrat, North…

PharmaceuticalPoliticsRare diseasesRegulationUSA

Novartis' Signifor approved in EU for acromegaly patients

Novartis' Signifor approved in EU for acromegaly patients

25-11-2014

The European Commission has approved Swiss drug major Novartis’ Signifor (pasireotide) for patients…

NovartisPharmaceuticalRare diseasesRegulationSigniforSwitzerland

GlaxoSmithKline files for added Revolade indication in EU

12-11-2014

UK pharma major GlaxoSmithKline has filed an application with the European Medicines Agency for a variation…

EuropeGlaxoSmithKlineLigand PharmaceuticalsOncologyPharmaceuticalPromactaRare diseasesRegulationRevolade

Sarepta will rebound strongly from eteplirsen US delay, says analyst

06-11-2014

US rare and infectious disease specialist Sarepta Therapeutics remains a strong contender in the Duchenne…

eteplirsenNeurologicalPharmaceuticalRare diseasesRegulationSarepta TherapeuticsUSA

Vertex seeks regulatory approval for lumacaftor/ivacaftor combination for CF

05-11-2014

US biotech firm Vertex Pharmaceuticals has filed for marketing approval for a fully co-formulated combination…

BiotechnologyEuropeivacaftorlumacaftorRare diseasesRegulationUSAVertex Pharmaceuticals

US FDA reveals discussions with Sarepta over eteplirsen

US FDA reveals discussions with Sarepta over eteplirsen

31-10-2014

In what is viewed as an unusual step, the US Food and Drug Administration made a website posting of its…

eteplirsenNeurologicalPharmaceuticalRare diseasesRegulationResearchSarepta TherapeuticsUSA

Scenesse recommended for rare disease by EMA/CHMP

Scenesse recommended for rare disease by EMA/CHMP

24-10-2014

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended…

Clinuvel PharmaceuticalsEuropePharmaceuticalRare diseasesRegulationScenesse

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

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