Kiadis Pharma receives FDA Regenerative Medicine Advanced Therapy (RMAT) des­ig­na­tion for ATIR101™

Amsterdam-Duivendrecht, The Netherlands, September 20, 2017 – Kiadis Pharma N.V. (“Kiadis Pharma” or the “Company”) (Euronext Amsterdam and Brussels: KDS), a clin­i­cal stage bio­phar­ma­ceu­ti­cal com­pa­ny devel­op­ing inno­v­a­tive prod­ucts to make bone mar­row trans­plan­ta­tions safer and more effec­tive for patients suf­fer­ing from blood can­cers and inher­it­ed blood dis­or­ders, today announces that the US Food and Drug Administration (FDA) has grant­ed ATIR101™, Kiadis Pharma’s lead inves­ti­ga­tion­al prod­uct for blood can­cers, the Regenerative Medicine Advanced Therapy (RMAT) des­ig­na­tion.
Arthur Lahr, CEO of Kiadis Pharma, com­ment­ed: “To receive the RMAT des­ig­na­tion from the FDA is an impor­tant mile­stone for Kiadis Pharma and a recog­ni­tion by the FDA of the sig­nif­i­cant poten­tial for ATIR101™ to help patients receive safer and more effec­tive bone mar­row trans­plan­ta­tions. RMAT is anal­o­gous to the Breakthrough Therapy des­ig­na­tion, and a clear val­i­da­tion of ATIR101™ towards doc­tors and investors. We are now going to work even clos­er with the FDA to agree a path to make this cell ther­a­py treat­ment avail­able for patients in the US as soon as pos­si­ble. In Europe ATIR101™ was filed for reg­is­tra­tion in April 2017 and we con­tin­ue to pre­pare the Company for the poten­tial European launch in 2019.
The RMAT path­way is anal­o­gous to the Breakthrough Therapy des­ig­na­tion designed for tra­di­tion­al drug can­di­dates and med­ical devices, and was specif­i­cal­ly cre­at­ed by the US Congress in 2016 to get impor­tant new cell ther­a­py and advanced med­i­c­i­nal prod­ucts to the patient ear­li­er. Just like the Breakthrough des­ig­na­tion, it allows com­pa­nies devel­op­ing regen­er­a­tive med­i­cine ther­a­pies to inter­act with the FDA more fre­quent­ly in the clin­i­cal test­ing process, and RMAT-des­ig­nat­ed prod­ucts may be eli­gi­ble for pri­or­i­ty review and accel­er­at­ed approval.
A regen­er­a­tive med­i­cine is eli­gi­ble for the RMAT des­ig­na­tion if it is intend­ed to treat, mod­i­fy, reverse or cure a seri­ous or life-threat­en­ing dis­ease or con­di­tion, and pre­lim­i­nary clin­i­cal evi­dence indi­cates that the drug has the poten­tial to address unmet med­ical needs for such a dis­ease or con­di­tion.
For more infor­ma­tion on RMAT des­ig­na­tion, vis­it the FDA web­site:
https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ucm537670.htm).
FDA has already request­ed an addi­tion­al meet­ing with Kiadis Pharma and will now work close­ly with the Company to pro­vide guid­ance on the sub­se­quent devel­op­ment of ATIR101™ for improved over­all sur­vival and reduced trans­plant relat­ed mor­tal­i­ty for patients receiv­ing a hap­loiden­ti­cal hematopoi­et­ic stem cell trans­plan­ta­tion (HSCT).

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