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FDA approves Vimizim to treat rare congenital enzyme disorder

FDA approves Vimizim to treat rare congenital enzyme disorder

17-02-2014

The US Food and Drug Administration on Friday (February 14) approved BioMarin Pharmaceutical’s Vimizim…

BioMarin PharmaceuticalNorth AmericaPharmaceuticalRare diseasesRegulationUSAVimizim

Retrophin to acquire Manchester Pharma in $62.5 million deal

Retrophin to acquire Manchester Pharma in $62.5 million deal

13-02-2014

USA-based Retrophin has signed an agreement to acquire Manchester Pharmaceuticals, a privately-held specialty…

Cardio-vascularChenodalManchester PharmaceuticalsMergers & AcquisitionsPharmaceuticalRare diseasesRetrophinVecamyl

Sanofi renews alliance with Regulus and ups investment by $10 million

Sanofi renews alliance with Regulus and ups investment by $10 million

05-02-2014

US pharma company Regulus Therapeutics has renewed its strategic alliance with French drug major Sanofi…

FinancialFranceOncologyPharmaceuticalRare diseasesRegulus TherapeuticsResearchSanofiUSA

US FDA grants Edison’s EPI-743 orphan status for Friedreich's ataxia

US FDA grants Edison’s EPI-743 orphan status for Friedreich's ataxia

04-02-2014

Privately-held US pharma company Edison Pharmaceuticals has announced that the US Food and Drug Administration…

Edison PharmaceuticalsEPI-743PharmaceuticalRare diseasesRegulationUSA

“Breakthrough Therapy” designation for GlaxoSmithKline’s Promacta/Revolade for SAA

“Breakthrough Therapy” designation for GlaxoSmithKline’s Promacta/Revolade for SAA

03-02-2014

The US Food and Drug Administration has granted “Breakthrough Therapy” designation for UK pharma…

GlaxoSmithKlinePharmaceuticalPromactaRare diseasesRegulationRevolade

FDA approves Hetlioz, first treatment for non-24 hour sleep-wake disorder in blind people

FDA approves Hetlioz, first treatment for non-24 hour sleep-wake disorder in blind people

03-02-2014

The US Food and Drug Administration on Friday approved US drugmaker Vanda Pharmaceutical's Hetlioz (tasimelteon),…

HetliozNeurologicalPharmaceuticalRare diseasesRegulationVanda Pharmaceuticals

UCB launches online search for rare phenotypes

UCB launches online search for rare phenotypes

28-01-2014

Belgian drugmaker UCB (Euronext Brussels: UCB) has launched an online search to find rare phenotypes…

GeneticsGlobalPharmaceuticalRare diseasesResearchUCB

Sanofi and MAbImprove LabEx collaborate on rare lung disease

Sanofi and MAbImprove LabEx collaborate on rare lung disease

24-01-2014

French drug major Sanofi and MAbImprove Laboratoire d'Excellence (LabEx), coordinated by France’s Universite…

PharmaceuticalRare diseasesResearchRespiratory and PulmonarySanofi

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

BioMarin buys rights to Repligen preclinical compounds

23-01-2014

BioMarin Pharmaceuticals has entered into an agreement to purchase from fellow USA-based Repligen Corp…

BioMarin PharmaceuticalBiotechnologyLicensingRare diseasesRepligen

Eisai’s amatuximab gains orphan status in EU for malignant mesothelioma

Eisai’s amatuximab gains orphan status in EU for malignant mesothelioma

22-01-2014

The investigational monoclonal antibody amatuximab (development code: MORAb-009), has today been granted…

amatuximabEisaiEuropePharmaceuticalRare diseasesRegulation

Mexico backs marketing of Aegerion’s Juxtapid for HoFH

Mexico backs marketing of Aegerion’s Juxtapid for HoFH

20-01-2014

US emerging biotech firm Aegerion Pharmaceuticals says that the Mexican Federal Commission for the Protection…

Aegerion PharmaceuticalsJuxtapidMexicoPharmaceuticalRare diseasesRegulationSouth America

Alexion inks deal with Moderna for rare diseases; “deal of the year” say analysts

Alexion inks deal with Moderna for rare diseases; “deal of the year” say analysts

15-01-2014

US drugmaker Alexion Pharmaceuticals has signed an exclusive strategic agreement with privately-held…

Alexion PharmaceuticalsLicensingModerna TherapeuticsPharmaceuticalRare diseases

GlaxoSmithKline returns Duchenne drug rights to Prosensa

GlaxoSmithKline returns Duchenne drug rights to Prosensa

13-01-2014

UK pharma giant GlaxoSmithKline says that its partner, Dutch biotech firm Prosensa has regained all rights…

BiotechnologydrisapersenGlaxoSmithKlineLicensingProsensaRare diseases

Genzyme to gain rights to certain Alnylam rare disease pipeline products

Genzyme to gain rights to certain Alnylam rare disease pipeline products

13-01-2014

French drug major Sanofi’s US biotech subsidiary Genzyme and Alnylam Pharmaceuticals have significantly…

Alnylam PharmaceuticalsBiotechnologyGenzymeLicensingMergers & AcquisitionspatisiranRare diseasesResearchSanofi

arGEN-X reaches two milestone payments in Shire deal and signs research agreement with Boehringer

13-01-2014

Belgium-based biotech firm arGEN-X has reached two important success milestones in its SIMPLE Antibody…

arGEN-XBelgiumBiotechnologyBoehringer IngelheimFinancialGeneticsLicensingRare diseasesResearchShire

Aegerion receives US subpoena over USA sales of Juxtapid

Aegerion receives US subpoena over USA sales of Juxtapid

13-01-2014

US biopharma company Aegerion Pharmaceuticals has received a subpoena from the US Department of Justice…

Aegerion PharmaceuticalsFinancialJuxtapidLegalPharmaceuticalRare diseasesUSA

Forest Labs to spend $2.9 billion on acquiring Aptalis

Forest Labs to spend $2.9 billion on acquiring Aptalis

09-01-2014

Confirming recent speculative reports, US drugmaker Forest Laboratories says it has entered into a definitive…

AptalisForest LaboratoriesGastro-intestinalsMergers & AcquisitionsPharmaceuticalRare diseases

moksha8 files Viibryd and Colobreathe in Brazil; amends Forest alliance

06-01-2014

Brazil-based pharma firm moksha8 has filed applications with the Brazilian regulatory authority, ANVISA,…

BrazilColoBreatheForest LaboratoriesLicensingMerck SeronoMoksha8NeurologicalPharmaceuticalRare diseasesRegulationSouth AmericaViibryd

Orphan designation for NPS Pharma’s Natpara in Europe

04-01-2014

The European Commission has granted orphan drug designation to US biotech firm NPS Pharmaceuticals Natpara…

BiotechnologyNatparaNPS PharmaceuticalsRare diseasesRegulation

Agilis Biotherapeutics and Intrexon collaborate on rare genetic disease

01-01-2014

US synthetic biology companies Agilis Biotherapeutics and Intrexon have entered an exclusive channel…

Agilis BiotherapeuticsBiotechnologyBoardroomFinancialIntrexonManagementRare diseasesResearch

Brazil’s ANS expands mandatory coverage for 29 genetic diseases

Brazil’s ANS expands mandatory coverage for 29 genetic diseases

26-12-2013

The Brazilian National Health Agency has authorized the criteria for the appropriate use of technology…

BrazilHealthcarePharmaceuticalRare diseasesSouth America

FDA approves Novo Nordisk’s Tretten to treat rare genetic clotting disorder

FDA approves Novo Nordisk’s Tretten to treat rare genetic clotting disorder

24-12-2013

The US Food and Drug Administration yesterday (December 23) approved Tretten, coagulation factor XIII…

HematologyNorth AmericaNovo NordiskNovoThirteenPharmaceuticalRare diseasesRegulationTretten

Italy’s Gentium accepts $1 billion takeover offer from Jazz Pharma

20-12-2013

Ireland-headquartered drugmaker Jazz Pharmaceuticals has entered into a definitive agreement to acquire…

defibrotideDefitelioGentiumItalyJazz PharmaceuticalsMergers & AcquisitionsNephrology and HepatologyPharmaceuticalRare diseases

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