NICE approves life changing gene therapy for treating spinal muscular atrophy

8 March 2021 - A new and potentially curative one-off gene therapy for babies with the rare genetic disorder spinal muscular ...

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Thousands of breast cancer patients to have routine access to NICE-approved drug combination

26 February 2021 - Another potentially life-extending drug combination for some people with advanced breast cancer will now be available for ...

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Emgality (galcanezumab) now approved for use in Canada for the treatment of episodic cluster headache

25 February 2021 - The first and only anti-CGRP treatment with dual indications. ...

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BioCryst receives positive CHMP opinion for Orladeyo (berotralstat), an oral, once daily therapy to prevent attacks in patients with hereditary angioedema

25 February 2021 - BioCryst Pharmaceuticals today announced that the CHMP of the EMA has adopted a positive opinion recommending ...

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Medexus announces expanded availability of Gleolan in Canada

25 February 2021 - Medexus Pharmaceuticals announced today that it has initiated the commercial launch of Gleolan in Canada, which had ...

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Empirical Spine initiates PMA submission process for FDA approval of Limiflex for degenerative spondylolisthesis

25 February 2021 - The LimiFlex Paraspinous Tension Band is a new, investigational device designed as an alternative to spinal fusion ...

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Retrotope granted rare paediatric disease designation from FDA for lead development candidate, RT001, in two life-threatening neurodegenerative indications

25 February 2021 - RT001 also granted fast track designation by FDA in Friedreich’s ataxia. ...

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NHS patients to benefit from first full access deal in Europe for new CAR-T therapy recommended by NICE

25 February 2021 - NICE has agreed a managed access agreement, via the Cancer Drugs Fund with manufacturer Kite, so more ...

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Allergan submits new drug application for investigational AGN-190584 for the treatment of presbyopia

25 February 2021 - If approved, this is expected to be the first eye drop to treat presbyopia. ...

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Exelixis announces breakthrough therapy designation granted to cabozantinib for the treatment of patients with previously treated radioactive iodine-refractory differentiated thyroid cancer

25 February 2021 - Exelixis expects to submit supplementary New Drug Application in 2021. ...

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FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation

25 February 2021 - Today, the U.S. FDA granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular ...

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NICE draft guidance recommends sapropterin for children with rare inherited metabolic condition phenylketonuria

25 February 2021 - NICE has today issued draft guidance for public consultation which recommends sapropterin (Kuvan, BioMarin) for treating phenylketonuria ...

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Adapting COVID-19 vaccines to SARS-CoV-2 variants: guidance for vaccine manufacturers

25 February 2021 - EMA has issued guidance outlining the requirements for manufacturers planning to modify their COVID-19 vaccines in ...

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International cooperation to align approaches for regulation of COVID-19 vaccines and medicines

25 February 2021 - Regulators around the globe are committed to aligning their regulatory requirements and addressing knowledge gaps to facilitate ...

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The common medicines hit with shortages as COVID supply issues worsen

24 February 2021 - Australians are being forced to take desperate measures to track down prescription medications they need as ...

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Three steps can help companies speed FDA approval of new drugs

24 February 2021 - Drug developers see FDA approval as a difficult uphill climb, requiring large investments of resources and ...

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