FDA Issues Complete Response Letter for KyndrisaTM for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Marketing Application in Europe Remains Under Review

SAN RAFAEL, Calif., Jan. 14, 2016 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the U.S. Food and Drug Administration (FDA) issued a Complete Response let­ter to the Company’s New Drug Application (NDA) for KyndrisaTM (dris­apersen) for the treat­ment of Duchenne mus­cu­lar dys­tro­phy (Duchenne) amenable to exon 51 skip­ping.
The FDA issues Complete Response let­ters to indi­cate that the review cycle for an appli­ca­tion is com­plete and that the appli­ca­tion is not ready for approval in its present form. FDA has con­clud­ed that the stan­dard of sub­stan­tial evi­dence of effec­tive­ness has not been met. BioMarin is review­ing the Complete Response Letter and will work with the FDA to deter­mine the appro­pri­ate next steps regard­ing this appli­ca­tion.
Duchenne affects approx­i­mate­ly 1 in every 3,500-5,000 male chil­dren, mak­ing it the most com­mon fatal genet­ic dis­or­der diag­nosed in child­hood. There is cur­rent­ly no FDA-approved ther­a­py designed specif­i­cal­ly to treat Duchenne.
The ongo­ing Kyndrisa exten­sion stud­ies will con­tin­ue, as will the ongo­ing clin­i­cal tri­als for oth­er exon-skip­ping oligonu­cleotides, BMN 044, BMN 045 and BMN 053, while BioMarin is explor­ing next steps for this appli­ca­tion. Patients cur­rent­ly receiv­ing Kyndrisa, BMN 044, BMN 045 and BMN 053 will remain on ther­a­py.

Kyndrisa Marketing Authorization Application Remains Under Regulatory Review in Europe

An appli­ca­tion for mar­ket­ing approval of Kyndrisa is also under review in the European Union. BioMarin antic­i­pates that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will pro­vide an opin­ion for the company’s Marketing Authorization Application (MAA) for Kyndrisa for the treat­ment of Duchenne mus­cu­lar dys­tro­phy (Duchenne) amenable to exon 51 skip­ping in the first half of 2016. If the CHMP opin­ion is pos­i­tive, the MAA will be referred to the European Commission (EC). If the MAA is approved by the EC, BioMarin would receive mar­ket­ing autho­riza­tion for Kyndrisa in all EU Member States. The EC is expect­ed to ren­der a final deci­sion for Kyndrisa in the sec­ond half of 2016.

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