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OxThera gains $10 million investment to continue Phase II trial

16-04-2014

Shareholders of privately-held Swedish biopharmaceutical company OxThera have agreed with a syndicate…

BiopharmaceuticalFinancialKidney diseasesOxabactOxTheraPharmaceuticalPrimary hyperoxaluriaRare diseasesRemi DrollerSweden

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

Australia to review program for life-saving drugs access

Australia to review program for life-saving drugs access

10-04-2014

The Australian government says that it will review the Life Saving Drugs Program (LSDP) - a move aimed…

Asia-PacificAustraliaFinancialHealthcarePharmaceuticalRare diseases

Promoting competition key to improving patients’ access to high cost medicines, says PHARMAC

Promoting competition key to improving patients’ access to high cost medicines, says PHARMAC

08-04-2014

New Zealand’s Pharmaceutical Management Agency, PHARMAC, has announced its intention to test out a…

Asia-PacificFinancialHealthcareNew ZealandPharmaceuticalRare diseases

EMA and Australia’s TGA collaborate in the area of orphan medicines

EMA and Australia’s TGA collaborate in the area of orphan medicines

07-04-2014

During a meeting last week at the European Medicines Agency, the EMA’s executive director, Guido Rasi,…

AustraliaEuropePharmaceuticalRare diseasesRegulation

UCB enters strategic alliance with Weill-Cornell in three discovery projects

03-04-2014

Belgian drugmaker UCB has launched a major, three-year strategic research alliance with Weill-Cornell…

MetabolicsPharmaceuticalRare diseasesResearchUCB

SOBI assumes direct responsibility for rare disease drug Orfadin in the USA and Canada

SOBI assumes direct responsibility for rare disease drug Orfadin in the USA and Canada

01-04-2014

Biotech firm Swedish Orphan Biovitrum has assumed direct responsibility for the distribution of its proprietary…

BiotechnologyMarkets & MarketingNorthern EuropeOrfadinRare diseasesSobi

Jazz Pharma and Gentium debut Defitelio in Europe

31-03-2014

Ireland-headquartered Jazz Pharmaceuticals and its Italian subsidiary Gentium have initiated the European…

AustriaDefitelioEuropeGentiumGermanyJazz PharmaceuticalsMarkets & MarketingPharmaceuticalRare diseases

Breakthrough therapies for rare diseases command premium pricing

Breakthrough therapies for rare diseases command premium pricing

26-03-2014

The global orphan drugs market presents plenty of opportunities for new drug development – while there…

PharmaceuticalPricingRare diseasesResearch

Promising mid-stage results for Prosensa’s drisapersen in DMD boys

19-03-2014

Shares of Dutch biotech firm Prosensa Holding rose 3.85% to $7.02 after the firm reported encouraging…

BiotechnologydrisapersenGlaxoSmithKlineProsensaRare diseasesResearch

More positive Ph III data for Amgen’s evolocumab

More positive Ph III data for Amgen’s evolocumab

17-03-2014

Leading independent biotech firm Amgen has released more positive clinical trial data with its cholesterol…

AmgenBiotechnologyCardio-vascularevolocumabRare diseasesResearch

Six-year follow-up data from Glybera treated patients

Six-year follow-up data from Glybera treated patients

17-03-2014

Netherlands-based human gene company uniQure and privately-held Italian drugmaker Chiesi Farmaceutici…

BiotechnologyChiesi FarmaceuticiGlyberaRare diseasesResearchuniQure

Marina Biotech to use its therapeutics platform to develop drugs for rare diseases

11-03-2014

US RNAi-based drug discover Marina Biotech says that it plans to broadly apply its RNAi, antisense and…

BiotechnologyFinancialMarina BiotechRare diseasesResearch

UK’s BIA finds government unwilling to set price cap for orphan disease treatment

UK’s BIA finds government unwilling to set price cap for orphan disease treatment

11-03-2014

The majority of Members of Parliament in the UK believe there should not be a maximum price per patient…

BiotechnologyPoliticsPricingRare diseasesUK

Ph III study for Jakavi meets endpoint in patients with polycythemia vera

Ph III study for Jakavi meets endpoint in patients with polycythemia vera

07-03-2014

Swiss pharma major Novartis today announced positive results from a pivotal Phase III trial of Jakavi…

IncyteJakaviNovartisOncologyPharmaceuticalRare diseasesRegulationResearch

Hawaii Biotech awarded $5.5million government grant for botulism therapy

Hawaii Biotech awarded $5.5million government grant for botulism therapy

05-03-2014

Privately-held US biotech company Hawaii Biotech has been awarded a contract to continue developing drugs…

BiotechnologyFinancialHawaii BiotechRare diseasesResearchUSA

ISIS Pharma earns $1 million from GSK for advancing ISIS-TTRRx

03-03-2014

USA-based Isis Pharmaceuticals has earned a $1 million milestone payment from UK pharma giant GlaxoSmithKline…

FinancialGlaxoSmithKlineIsis PharmaceuticalsISIS-TTRRxPharmaceuticalRare diseasesResearch

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

Which place for orphan drugs in the strategy of pharma companies and payers?

27-02-2014

As the 7th Rare Disease Day will be held on February 28, ALCIMED, an innovation and new business consulting…

Markets & MarketingNovartisPharmaceuticalPricingRare diseasesResearchRocheSanofi

EMA/CHMP recommends 12 new orphan medicines over the past year

26-02-2014

Over the past 12 months, a total of 12 medicines for the treatment of rare diseases were recommended…

EuropePharmaceuticalRare diseasesRegulation

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

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