Ann: FDA Grants Rare Pediatric Disease Designation for Revascor, page-139

@col69 Oh I don't think its small....criteria to obtain the designation admittedly less than orphan drug designation ordinarily, but the FDA still has to somewhat comfortable with some suggested efficacy of the drug or treatment, and it needs to be demonstrated in vivo:

"A rare pediatric disease is one that is serious or life-threatening in which the serious or life-threatening manifestations primarily affect patients from birth to 18 years, including neonates, infants, children, and adolescents. It must also be a rare disease or condition as described in the FD&C Act, with a prevalence of fewer than 200,000 people in the United States."

https://premierconsulting.com/resources/blog/fda-designations-for-rare-disease-products-part-3-rare-pediatric-disease-designation/#Criteria-to-obtain-the-designation

"There must be supportive data suggesting that the drug may be effective in the rare pediatric disease or condition."

Let alone the valuation of the voucher in other Revascor application approvals or any monetary value which MSB might seek to exploit down the line or the effects this will have towards sentiment or analysts assessment of its approval and subsequent commercialisation likelihood, which has taken blow after blow...

Many investors dumped MSB for simply believing the FDA was against MSB's science and approach. The FDA at least believes, and this suggests it definitely believes, it did so on the merit of MSB's applications to date, which bodes well for the accelerated approval ambitions of Silviu et al in Revascor for CHF and indeed approval ambitions in Remestemcel-L under its new GvHD BMT CTN trial partnership too. I suspect any prospective CHF partners will be far less irrationally predisposed to any such ideas of the FDA having it in for MSB now.

(Forgive me for spelling out the bleeding obvious, but other posters assume some of us do not know if I do not )

and for those not familiar with the benefits:


(they've actually gone for the $350 million as suggested in other posts prior to 2017).