BioMarin Discontinues Clinical Development of Drisapersen

Today is June 1 and I know many of us feel like 2016 has beat up this community already. With every small victory, we face another major setback.


As you have heard by now, BioMarin announced that they were discontinuing the clinical development of drisapersen after interactions with both the FDA and EMA. We are heartbroken.


To say Duchenne is difficult is a gross understatement. Until you have lived it, you have no idea how challenging it is for even the strongest of us. Any parent would do anything they could to help their child. But I think the sacrifices we make as families living with Duchenne are exceptional and many.


Families contribute their child, their time, their finances, their quality of life for a chance at something different, something better. We sit through meetings, webinars, read the latest/greatest articles, press releases, listen carefully to the online chatter, everyone speculating what they think is happening. At the end of the day, given the lifespan of Duchenne and the narrow inclusion criteria, it is likely that we will only have one shot on goal and the 'shot' is carefully considered and guessed to be the single chance for something different, better. Something that translates into stabilization for a period of time, meaning walking longer, standing longer, feeding – just lifting that damn fork to the mouth – breathing, and living one more day than predicted. And sometimes it is the ONLY shot possible in a lifetime, given the years it takes.  


BioMarin’s decision is not entirely unexpected based on what we learned over time and detailed in the advisory committee meeting. But unexpected is different than unanticipated...we were all hopeful it would work and we saw, that for many, it did.  We were hopeful for future developments, additional exons, possibilities for many.

We will be talking to BioMarin and asking for clarification of next steps. In their press release, they said will continue to explore the development of next generation oligonucleotides for the treatment of Duchenne. We will ask what this means and we will report back to you.


I think we all know that drug development is difficult,  that 1:8 drugs are finally approved. We could recite statistics in our head, much like the statistics for Duchenne. But when those statistics become your own, worn on your heart, and carried in the backpack of our lives, hearts are broken and statistics no longer matter. Those statistics are the lives of our loved ones, lives turned upside down and inside out.


While it feels like the rug has been pulled out again and again, PPMD continues to work with industry partners, including BioMarin, so that our full and promising pipeline doesn’t lose momentum. There is good news out there and there is progress being made. There is hope.


For the hundreds of young men and their families who have participated in the drisapersen trial for years now, your sacrifice is not in vain. You have changed the landscape of drug development and you are heroes of this community. “Thank you” is simply not enough. We will be working day and night for more shots on goal for you and all those diagnosed.


There is so much more work to do and PPMD is here to do it. We will let you know as new information becomes available. 


We will not stop until we end Duchenne for every single person in this community.

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Comment by ramon ciriaco viaña ortiz on July 5, 2016 at 11:10am

Hola  como  estan  grasias  por  dejar entra a su comunidad un  comentario serca de mii hijo con  duchenne, a  el  lo estaba atendiendo una neurologa  aca en  Cartagena  Colombia de nombre  MIRIAN BARBOSA la  dre queria  prestarle  todas  las  herramientas nesesaria  por  su  emfermedad  pero  ya  salio  pensionada he perdido  todo  contacto con  ella, hase  mas  de  6  meses  a  mi  hijo  le tomaron  una  muestras  laboratorios  por  intermedios  de  su  laboratorio ,  no se  si son contratados por  ustedes  el  laboratorio que da a ca en  cartagena en el centro comercial  la plasuela,  nosotros  todavia  no  tenemos  esos  resultados  y  no  sabemos  conque   fin   fueron  tomadas  esas  pruevas  de  laboratorios  no  se  ustedes  saben  algo  de  esto  estos  son  los  datos  de  mi  hijo....nombre  JOSE DAVID  VIAÑA  DIAZ   identificacion  1007639651,,,, estos son  mis contastos  ,,,telefono  6695  celular  3004117898   correo   :   La  muestra  no   si  era  para  algun  medicamento    POR  FAVOR  COLABORENME  LO  MAS  PRONTO  ATTMT.  rAMONNcIRIACO  VIAÑA Ortiz.........

Comment by Shelly on June 1, 2016 at 11:56am
Thanks Pat for all the support!!

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