Top Tweets for #ValRox
Well, that’s a first!
My name is listed on a scientific poster in @nbd_foundation’s Exhibit Hall!
Proud moment, to be included w such a list of heavyhitters in alignment on the framing & language used to communicate about #ValRox #GeneTherapy to a broader #hemophilia audience.
valrox、頭打ちの兆し
#Biomarin の #血友病A遺伝子治療薬 #valoctocogene_roxaparvovec/#valrox が第VIII因子活性を3年後も顕著に抑制した。しかし、かつては1回で終わると言われていたのが、今は5年以上���続する見込みであると話していることは注目に値する。
https://t.co/KuIoaYj3Qy
#Evaluate
Q: any risk to the #Valrox filing based
on these $PKU safety concerns?
$BMRN says: "given the #FDA's recent track record in
conservatism, they're #not #unreasonable questions and just I don't think #anybody really #knows"
EVERCORE $BMRN "a little uneasy
about readthrough from the preclinical #oncogenic signal seen with the PKU #AAV5 gene therapy.
With the FDA now demanding additional analysis for that program, it could impact
#Roctavian’s review."
Key questions: How many patients needed steroids, what was the variability in levels (and how many had levels <10% and <5%), how long is the response predicted to last? #hemophilia #genetherapy #ValRox 2/4
$BMRN BioMarin International Limited #withdrew its application for a marketing authorisation of
Roctavian ( #VALROX )
for the treatment of severe #hemophilia A (haemophilia A)
anothe VE+ $SGMO $PFE
https://t.co/9ubyv2AMII
$BMRN Biomarin Receives Complete Response Letter From FDA For #roctavian AKA #VALROX Valoctocogene Roxaparvovec Gene Therapy
V++++++++++++++ $SGMO $PFE
#Roctavian (#Valoctocogeneroxaparvovec or #valrox) will likely draw patients both from those currently treated with #Hemlibra (29%) and those currently treated with Factor VIII prophylaxis $BMRN $RHHBY $SGMO -Hemophilia A Gene Therapy- KOLs Insight-2020
https://t.co/opXqUg9tYv
Some big #Pdufa decisions due soon, my look at what August has in hold via @evaluatevantage, @evaluatepharma data: https://t.co/oB5YYZFPI1 $BMRN $RHHBY $GILD $GLPG $NVS #valrox #risdiplam #filgotinib #inclisiran #FDA
GoldmanSachs: highlight $BMRN as a laggard that we expect to move (4-year #valrox data in mid-year serving as an overhang)
Options imply $BMRN (8%; sheltered from covid disruption though 4-year durability data expected in #May )
$BMRN earnings 4/29
#hemophilia
Wedbush puts $BMRN on best idea list
Based on the #Valrox clinical dataset, we anticipate approval and project annual sales could reach over *** $2BN *** in 2024 after our U.S. estimated launch ~YE:20, EU
launch in Q2:21 and RoW launches starting in Q4:21.
RE $SGMO hemophila $PFE
New drugs enter the market every year, but only a few will achieve blockbuster status with annual sales of $1bn or more. The new #DrugstoWatch2020 list from @Cortellis & @BioWorld identifies 11 #blockbuster drugs: https://t.co/zflCzQbPj4
#Rimegepant #Ozanimod #Valrox #Vadadustat
$BMRN #VALROX BioMarin's Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by FDA with Review Action Date of August 21, 2020
If approved, Biomarin’s ($BMRN) #Valrox would be the first potentially curative approach to hemophilia A, eliminating the need for blood transfusions and FVIII replacement therapy after a single infusion. @Cortellis #drugstowatch2020 list $BMRN https://t.co/vRJetSyKIO
Biomarin’s ($BMRN) #Valrox is one of the 11 potential blockbuster drugs in @Cortellis #drugstowatch2020 report. The one and done gene therapy for #hemophilia A could be approved by FDA in July and be priced at $2.8M $BMR https://t.co/vRJetSyKIO
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