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Valrox
This page shows the latest Valrox news and features for those working in and with pharma, biotech and healthcare.
Presidential hopeful Bernie Sanders criticises BioMarin’s gene therapy price
The new therapy, Valrox (valoctocogene roxaparvovec), improved levels of clotting Factor VIII sufficiently in eight of 20 adults with severe haemophilia A treated over three years in BioMarin’s phase 3 ... Now, BioMarin is eyeballing a $2m-$3m
Latest news
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Sangamo steps up BioMarin chase with haemophilia gene therapy data
Sangamo is adamant that it is still in contention in the haemophilia A gene therapy market, despite BioMarin’s stated plan of filing for approval of its valoctocogene roxaparvovec or ‘valrox’ ... Three-year data on its candidate were reported in May
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Roche’s Hemlibra scores broader NHS funding
BioMarin is in pole position with its candidate valrox, which it plans to submit by the end of 2019.
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BioMarin ticks over in Q2 ahead of gene therapy filing
The game-changer for the company, however, is its gene therapy for haemophilia A, valoctocogene roxaparvovec, known as valrox. ... valrox and vosoritide, its late stage candidate for achondroplasia, (the most common form of dwarfism) which they say have
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ACPs to lead gene therapies in haemophilia market growth
BioMarin’s ValRox, which is expected to launch as early as 2020, and Spark Therapeutics’ SPK-8011 for haemophilia A, and uniQure’s AMT-061 and Pfizer/Spark Therapeutics’ SPK-9001 ... Therefore, first-to-market advantage will drive commercial
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New results back Sangamo, UniQure haemophilia gene therapies
It’s a case of so far, so good with SB-525 as Sangamo and Pfizer set off in pursuit of BioMarin’s valrox candidate, which is already in a phase
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Keeping up with the developing regulatory requirements for gene and cell therapies
The FDA’s requirement on Biomarin to provide an additional two years of clinical data of Valrox before approval signals an increase in regulatory scrutiny for the regenerative medicine industry.
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Cortellis Drugs to Watch in 2020 report
Valrox is in regulatory review in the US, with a PDUFA date of 21 August 2020, with an MAA also being reviewed in the EU. ... For this reason, even if the penetration is modest, Valrox could post significant revenue.
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