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[Andy Battaglia]

Great news today! The new drug, REBLOZYL (Luspatercept) has been approved by the FDA today.

https://ir.celgene.com/press-releases/press-release-details/2019/FDA-Approves-REBLOZYL-luspatercept-aamt-for-the-Treatment-of-Anemia-in-Adults-With-Beta-Thalassemia-Who-Require-Regular-Red-Blood-Cell-Transfusions/default.aspx?fbclid=IwAR04eCT3kPgz4ysLirPWdIjmoFHGAWuu7omfB2hOT4ahZNct0U4r9s88lDw

REBLOZYL is the first and only FDA-approved erythroid maturation agent, representing a new class of therapy which works by regulating late-stage red blood cell maturation to help patients reduce their RBC transfusion burden.
REBLOZYL is anticipated to be available [in the US] 1 week following the FDA approval.

At this point, I do not know when it will become available outside the US, but I will try to inform once I know more.

[Sunny2701]

Great news! First Gene therapy approval and now this. This definatly is a big step forward. Let's hope everyone is able to reap benefits of these new modes of treatments as early as possible. Although this may not get us totally off transfusions, but there is a silver lining:)

[anshul]

Great News!!

Do we know if only Adults or Kids also can be benefited once it is available?

[Emby]

Thank you for this great news Andy!
Please can you help me to understand how exactly it works, although you have highlighted it.
I thought the Hb weakens when blood cells mature. Sorry mind fog. Will it be in medicine form?

Thank you and sorry just can't seem to think straight at the moment.

[Sunny2701]

Reblozyl will be available commercially in one week and will cost $3,441 per 25 mg vial at list price, the companies said. The drug is dosed by weight, typically at a dose of 1 milligram per kilogram.

For the recommended once per every three weeks regimen, that works out to an annual cost over $170,000 — "significantly higher" than expected, according to SVB Leerink analyst Geoffrey Porges.

Ref- https://www.biopharmadive.com/news/celgene-win-fda-approval-luspatercept-reblozyl-beta-thalassemia/566968/

[Vikramjit Singh]

Great news!

[Andy Battaglia]

Not yet approved for children.

https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761136lbl.pdf

8.4 Pediatric Use
Safety and effectiveness in pediatric patients have not been established. Based on findings in
juvenile animals, REBLOZYL is not recommended for use in pediatric patients [see NonClinical Toxicology (13.1)].

[Andy Battaglia]

Emby,

Reblozyl is an erythroid maturation agent. That means that it takes immature red blood cells that would not normally mature in thalassemics and it causes them to mature, adding to the RBC total and the Hb level. It is given as a subcutaneous injection every few weeks.

[smit-17]

How many % reduction  in transfusion burden
For β0/β0- major?