BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa™ (dris­apersen) in Europe

SAN RAFAEL, Calif., May 31, 2016 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has with­drawn its Kyndrisa™ (dris­apersen) Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) fol­low­ing dis­cus­sions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meet­ing. Those dis­cus­sions clear­ly indi­cat­ed that the CHMP intend­ed to issue a neg­a­tive opin­ion. Kyndrisa is an exper­i­men­tal drug for the treat­ment of Duchenne mus­cu­lar dys­tro­phy (DMD) amenable to exon 51 skip­ping.
Based on dis­cus­sions at the CHMP meet­ing and the Food and Drug Administration Complete Response Letter in January, BioMarin intends to dis­con­tin­ue clin­i­cal and reg­u­la­to­ry devel­op­ment of Kyndrisa as well as the three oth­er first-gen­er­a­tion fol­low-on prod­ucts, BMN 044, BMN 045 and BMN 053, cur­rent­ly in Phase 2 stud­ies for dis­tinct forms of Duchenne mus­cu­lar dys­tro­phy. Notwithstanding this out­come for Kyndrisa in Europe, the Company con­tin­ues to expect to achieve non-GAAP break-even or bet­ter in 2017.
BioMarin plans to work with physi­cians, patient groups, and reg­u­la­to­ry author­i­ties to devel­op a tran­si­tion plan for those patients cur­rent­ly being treat­ed with Kyndrisa, BMN 044, BMN 045 and BMN 053. The Company will con­tin­ue to explore the devel­op­ment of next gen­er­a­tion oligonu­cleotides for the treat­ment of Duchenne mus­cu­lar dys­tro­phy.
The with­draw­al of the MAA and dis­con­tin­u­a­tion of our cur­rent exper­i­men­tal drugs for Duchenne is a dif­fi­cult but nec­es­sary deci­sion at this time,” said Jean-Jacques Bienaimé, BioMarin chair­man and chief exec­u­tive offi­cer. Mr. Bienaimé added, “We want to extend our sin­cere grat­i­tude to all of the fam­i­lies and care­givers who sup­port­ed our efforts over the last year to bring Kyndrisa to patients with Duchenne. Our plan now is to invest in research of next gen­er­a­tion oligonu­cleotides with the goal of mak­ing a safe and effec­tive treat­ment avail­able for boys with this dev­as­tat­ing dis­or­der.

Leave a Reply

Your email address will not be published. Required fields are marked *