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Catalyst Pharma updates on Ph III Firdapse study in LEMS

13-08-2014

US rare diseases focussed biotech firm Catalyst Pharmaceutical Partners provided an update on the progress…

BiotechnologyCatalyst Pharmaceutical PartnersFirdapseLambert-Eaton myasthenic syndromeRare diseasesRegulationResearchTreatment of Lambert-Eaton myasthenic syndromeUSA

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

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

Vertex leaps on positive Ph III results with lumacaftor

Vertex leaps on positive Ph III results with lumacaftor

24-06-2014

US biotech firm Vertex Pharmaceuticals saw its share price rocket nearly 52% to $101.20 in extending…

BiotechnologyBusiness FinanceCystic fibrosisHealth Medical PharmaKalydecolumacaftorMedicinePhenolsRare diseasesRegulationResearchVertex Pharmaceuticals

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

BioLineRx gains orphan status for novel stem cell mobilization treatment

BioLineRx gains orphan status for novel stem cell mobilization treatment

23-01-2014

Israeli drug developer BioLineRx has received notice from the US Food and Drug Administration confirming…

BioLineRxBiotechnologyBL-8040Rare diseasesRegulationResearch

Sarepta plunges as FDA questions eteplirsen trial results

Sarepta plunges as FDA questions eteplirsen trial results

12-11-2013

Shares of US RNA-based drug developer Sarepta Therapeutics plummeted in pre-market trading on November…

BiotechnologydrisaperseneteplirsenGlaxoSmithKlineNorth AmericaRare diseasesRegulationResearchSarepta Therapeutics

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

FDA grants orphan designation for Armagen's lead product

19-07-2013

California, USA-based Armagen says that the US Food and Drug Administration has granted orphan drug designation…

AGT-182ArmagenPharmaceuticalRare diseasesRegulationResearch

Celgene gets negative NICE view on Revlimid in rare blood cancer; presents new data in newly-diagnosed MM

11-07-2013

US biotech firm Celgene (Nasdaq: CELG) today (July 11) expressed disappointment at the preliminary decision…

BiotechnologyCelgeneEuropeOncologyPricingRare diseasesRegulationResearchRevlimid

The case for Insmed's CF drug candidate Arikace; analysts' views

05-07-2013

The 16% decline in US biotech firm Insmed's (Nasdaq CM: INSM) stock is an overreaction to results from…

ArikaceBiotechnologyFinancialInsmedRare diseasesRegulationResearchRespiratory and Pulmonary

New idiopathic pulmonary fibrosis treatment may open up valuable research opportunity, says GlobalData

17-04-2013

Further research is a prerequisite to shine a light on idiopathic pulmonary fibrosis (IPF) and offer…

BiotechnologyEsbrietInterMunePricingRare diseasesRegulationResearch

New Canadian rare disease strategy welcomed as offering hope and solutions for patients

04-10-2012

The Canadian government this week announced a new national framework to improve access to "orphan drugs"…

BiotechnologyNorth AmericaPharmaceuticalRare diseasesRegulationResearch

Positive Ph III results for Biogen Idec and SOBI's hemophilia B agent

27-09-2012

US biotech firm Biogen Idec (Nasdaq: BIIB) and partner Swedish Orphan Biovitrum (STO: SOBI) have announced…

Biogen IdecBiotechnologyEuropeNorth AmericaPharmaceuticalRare diseasesRegulationResearchrFIXFcSobiSwedish Orphan Biovitrum

News briefs: US priority review for regorafenib; Ph IIb blisibimod data

29-06-2012

Bayer HealthCare, a US unit of Germany's Bayer (BAYN: DE) and partner Onyx Pharmaceuticals (Nasdaq: ONXX)…

Anthera PharmaceuticalsBayerBiotechnologyblisibimodOncologyOnyx PharmaceuticalsPharmaceuticalRare diseasesregorafenibRegulationResearch

Actelion drops setipiprant, gets miglustat approval in Japan

04-04-2012

Switzerland-based Actelion Ltd (SIX: ATLN) announced says that it will re-direct its development efforts…

ActelionAsia-PacificBiotechnologyBrazavesInflammatory diseasesMiglustatRare diseasesRegulationResearchsetipiprantZavesca

Vertex plans for Kalydeco launch

09-01-2012

US drugmaker Vertex Pharmaceuticals (Nasdaq: VRTX) announced its 2012 business objectives in conjunction…

Anti-viralsIncivekKalydecoPharmaceuticalRare diseasesRegulationResearchVertex

ImmuPharma’s Lupuzor OKed to start Phase III and fast-tracked by US FDA

03-11-2011

UK-based ImmuPharma plc (LSE:IMM), which last month regained rights to its investigational lupus drug…

ImmuPharmaLupuzorPharmaceuticalRare diseasesRegulationResearch

Novartis and Vectura’s NVA237 filed in Europe; positive Ph III data at ERS; Gilenya, Ilaris approved in Japan

28-09-2011

Shares of UK-based Vectura (LSE: VEC) rose 5.5% to 91 pence by midday yesterday, after the company announces…

Asia-PacificEuropeGilenyaIlarisNeurologicalNovartisPharmaceuticalRare diseasesRegulationResearchSeebriVectura

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