Regulation - Rare diseases


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GSK files for US approval for Promacta for severe aplastic anemia

GSK files for US approval for Promacta for severe aplastic anemia


UK pharma giant GlaxoSmithKline has submitted a supplemental New Drug Application to the US Food and…

GlaxoSmithKlineLigand PharmaceuticalsNorth AmericaOncologyPharmaceuticalPromactaRare diseasesRegulationUSA

US orphan status for GW Pharma’s Epidiolex in Lennox-Gastaut syndrome


UK-based GW Pharmaceuticals says that the Food and Drug Administration has granted orphan drug designation…

epidiolexGW PharmaceuticalsNeurologicalNorth AmericaPharmaceuticalRegulationUSA

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


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

Markets & MarketingNovartisPharmaceuticalPricingRare diseasesResearchRocheSanofi

Negative NICE appraisal for Eli Lilly’s Alimta as maintenance therapy


UK health care guidance body the National Institute for Health and Care Excellence (NICE) has published…

AlimtaEli LillyNorthern EuropeOncologyPharmaceuticalPricingRegulationUK

BIO urges Indiana Governor to sign Bill on interchangeable biologic medicines


US trade group the Biotechnology Industry Organization (BIO) and the Indiana Health Industry Forum (IHIF)…

BiosimilarsGenericsLegalNorth AmericaRegulationUSA

Cell Thera’s NHL drug Pixuvri gets backing from UK’s NICE


US biotech firm Cell Therapeutics has received positive news from the UK’s drugs watchdog for use of…

BiotechnologyCell TherapeuticsNorthern EuropeOncologyPixuvriPricingRegulationUK

UK’s NICE gives green light to Bayer’s Eylea in final guidance


UK drugs watchdog the National Institute for Health and Care Excellence (NICE) has published final guidance…

BayerEyleaNorthern EuropeOphthalmicsPharmaceuticalPricingRegulationUK

US FDA approves Anika’s Monovisc for osteoarthritis pain of the knee


US drugmaker Anika Therapeutics has received marketing approval for Monovisc from the US Food and Drug…

Anika TherapeuticsAnti-Arthritics/RheumaticsDePuyMonoviscNorth AmericaPharmaceuticalRegulationUSA

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


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


The US Food and Drug Administration has approved Myalept (metreleptin for injection) as replacement therapy…

AstraZenecaBristol-Myers SquibbmetreleptinMyaleptNorth AmericaPharmaceuticalRare diseasesRegulationUSA

Business community welcomes European Parliament vote on counterfeit goods in transit


A long list of trade organizations representing thousands of companies across all industry lines and…

EuropePatents & Trade marksPharmaceuticalRegulation

Iroko Pharma gains FDA approval for low-dose indomethacin drug Tivorbex


US specialty drugmaker Iroko Pharmaceuticals has received approval from the US Food and Drug has approved…

Iroko PharmaceuticalsNeurologicalNorth AmericaPharmaceuticalRegulationTivorbexUSA

US orphan drug market outlook 2018: Report


In the largest market for orphan drugs, the USA, there was a shortage of adequate therapies for treating…

Markets & MarketingNorth AmericaPharmaceuticalRare diseasesRegulationUSA

NICE guidance will deprive UK patients with newly-diagnosed advanced melanoma of Yervoy


US drug major Bristol-Myers Squibb today expressed its disappointment that the UK drugs watchdog the…

Bristol-Myers SquibbNorthern EuropeOncologyPharmaceuticalPricingRegulationUKYervoy

FDA proposed rule to change label requirements would cause dangerous confusion, raise costs


The Food and Drug Administration’s Proposed Rule to change prescription drug label requirements risks…

GenericsNorth AmericaRegulationUSA

FDA Breakthrough designation for all-oral daclatasvir for chronic hepatitis C


The US Food and Drug Administration has granted drug major Bristol-Myers Squibb’s investigational DCV…

Anti-viralsBristol-Myers SquibbdaclatasvirNorth AmericaPharmaceuticalRegulationUSA

Novartis gets fourth indication, DME, for Lucentis in Japan


Swiss drug major Novartis says it has received approval from Japanese regulatory bodies for a fourth…


IQWiG now finds “major” benefit for Xtandi


In an early benefit assessment under Germany’s Act on the Reform of the Market for Medicinal Products…

Astellas PharmaGermanyMedivationNorthern EuropeOncologyPharmaceuticalPricingRegulationXtandi

Indian drugmakers readying for next “Patent cliff”


The strategic drug reviews, as well as mergers and acquisitions – such as Actavis buying out Forest…

Asia-PacificGenericsIndiaMergers & AcquisitionsPatentsPharmaceuticalRegulation

FDA backs Kalydeco for eight additional mutations that cause cystic fibrosis


The US Food and Drug Administration approved biotech firm Vertex Pharmaceuticals’ supplemental New…

BiotechnologyKalydecoNorth AmericaRare diseasesRegulationUSAVertex

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