Pharmaceutical - Rare diseases

Filter

Current filters:

Rare diseases

Popular Filters

1 to 25 of 299 results

OPKO Health leaps on potential $570 million deal with Pfizer

OPKO Health leaps on potential $570 million deal with Pfizer

15-12-2014

US drugmaker OPKO Health saw its shares leap 13.8% in pre-market trading, when it revealed a licensing…

LicensingOPKO HealthPfizerPharmaceuticalRare diseasesUSA

Shire in $15 million research collaboration with CFFT on novel CF treatment; pipeline news

Shire in $15 million research collaboration with CFFT on novel CF treatment; pipeline news

10-12-2014

Cystic Fibrosis Foundation Therapeutics Inc, the non-profit drug discovery and development affiliate…

LifitegrastOphthalmicsPharmaceuticalRare diseasesResearchShireSHP607SHP625SHP626Vyvanse

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

Alizé Pharma acquires exclusive worldwide rights for new peptide to tackle bone diseases

03-12-2014

Alizé Pharma, a group of privately-held biopharmaceutical companies specialized in the development of…

Alize PharmaLicensingPharmaceuticalRare diseasesUSA

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

BioMarin to acquire Prosensa for up to $840 million

BioMarin to acquire Prosensa for up to $840 million

24-11-2014

US drugmaker BioMarin Pharmaceutical has entered into a definitive agreement to purchase all of the outstanding…

BioMarin PharmaceuticaldrisapersenMergers & AcquisitionsPharmaceuticalProsensaRare diseases

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

UPDATE: AstraZeneca divests Myalept for $325 million

06-11-2014

Anglo-Swedish pharma major AstraZeneca today announced that it has entered into a definitive agreement…

Aegerion PharmaceuticalsAstraZenecaMergers & AcquisitionsMyaleptPharmaceuticalRare diseases

AOP Orphan brings Thromboreductin to Russian market

04-11-2014

Austria-based rare diseases specialist AOP Orphan is expanding its sales area for Thromboreductin (anagrelide)…

AnagrelideAOP OrphanMarkets & MarketingPharmaceuticalRare diseasesRussiaThromboreductin

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

Orphan drug market forecast to reach $176 billion by 2020

30-10-2014

In 2020, orphan drugs are expected to account for 19% of the total share of prescription drug sales excluding…

Markets & MarketingPharmaceuticalRare diseases

Australian PBS listing for Kalydeco and Soliris

Australian PBS listing for Kalydeco and Soliris

27-10-2014

Kalydeco (ivacaftor) and Soliris (eculizumab) – new treatments for cystic fibrosis (CF) and hemolytic…

Alexion PharmaceuticalsAustraliaFinancialHealthcareKalydecoPharmaceuticalRare diseasesSolirisVertex Pharmaceuticals

FDA approves Baxter’s new treatment for rare form of hemophilia

FDA approves Baxter’s new treatment for rare form of hemophilia

25-10-2014

The US Food and Drug Administration on Friday approved Obizur (antihemophilic Factor [recombinant), porcine…

Baxter InternationalHematologyObizurPharmaceuticalRare diseasesUSA

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

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

Endo’s buy of struggling Auxilium for $2.6 billion analysed by GlobalData

12-10-2014

Endo International’s $2.6 billion purchase of Auxilium Pharmaceuticals (The Pharma Letter October 9)…

Auxilium PharmaceuticalsEndo InternationalMen's HealthMergers & AcquisitionsPharmaceuticalRare diseasesTestimTestopelXiaflex

BTG settles CroFab patent litigation with Bioclon and RDT

10-10-2014

UK specialist health care company BTG says it has settled its US patent infringement complaint against…

BTGCrofabInstituto BioclonLegalPatentsPharmaceuticalRare Disease TherapeuticsRare diseasesUSA

FDA grants to stimulate drug and device development for rare diseases

FDA grants to stimulate drug and device development for rare diseases

01-10-2014

The US Food and Drug Administration yesterday announced it has awarded 15 grants totaling more than $19…

FinancialPharmaceuticalRare diseasesResearchUSA

Avalon Ventures and GlaxoSmithKline launch two new companies

23-09-2014

Avalon Ventures today announced the launch of two new companies formed through its collaboration with…

Avalon VenturesFinancialGlaxoSmithKlineHematologyMergers & AcquisitionsPharmaceuticalRare diseasesSilarus TherapeuticsThyritope Biosciences

Akashi gets rights to novel DMD treatment from Tonus

Akashi gets rights to novel DMD treatment from Tonus

15-09-2014

US clinical-stage biopharma firm Akashi Therapeutics has acquired global rights to GsMTx-4, a small molecule…

Akashi TherapeuticsGlobalGsMTx-4LicensingPharmaceuticalRare diseasesTonus Therapeutics

Depomed wins in law suit seeking FDA orphan drug exclusivity for Gralise

Depomed wins in law suit seeking FDA orphan drug exclusivity for Gralise

07-09-2014

US specialty drugmaker Depomed says that Judge Kentaji Brown Jackson of the federal district court for…

DepomedGraliseLegalNeurologicalPharmaceuticalRare diseasesUSA

1 to 25 of 299 results

Back to top