I agree.Unmet need for small patient population. BLA excepted...

I agree.
Unmet need for small patient population.

BLA excepted after five weeks of assessment.

CRL answered all concerns.

4 inspectors in Singapore, 10 days no problem.

Assays reasonable.

MOA correlated to CRP levels.

Can’t do a RCT trial in kids.

1, 2, 4 year data survival 50%.

Extra EAP data - 247 kids.

Grade C - D salvage therapy. Incredible really. Kids will die otherwise. Last chance saloon.

Trumps all best available therapies.

Ruxolitinib approved after single arm trial.

Safety profile perfect, no side effects.

New gene & cell department created at FDA with focus on making accelerated approval easier to attain.

Ryoncil more potent then Prochymal.

EAP FDA approved. Why would they do that if it didn’t work? Shows that their main concern was batch to batch potency and consistency.

ARDS results 43% same MOA.

Phil and Greg loaded up.

Phil ex FDA 20 years of knowledge in approvals - back of the hand stuff.

Phil so good at GVHD submission he’s been given new role for heart approval - pretty obvious really.

30M worth of stock ready to go.

Staged roll out.

Sales team ready.

Parents praying for it.

Off label use.

Let’s go!

I have also noticed that an adult and chronic GVHD trial has been added to the pipeline. I’m tipping that will be announced as part of the approval in a couple of weeks.

It works - Real World Evidence

Used in Japan for GVHD since 2016 40% market share and growing.

Takeda licensed to use rem-l for Crohns complex perianal fistulas - Alofisel since 2017

Alofisel is approved in the European Union/European Economic Area, Israel, Switzerland and the United Kingdom for the treatment of complex perianal fistulas in adult patients

Takeda scaled up due to high demand of Alofisel. Invested 36M to build new manufacturing facility in Ireland to supply Crohn’s therapy to patients in Europe, the US and Canada

It works, it’s safe, it’s time.

And to think this is just the entrée.