FDA Advisory Committee votes “Yes” to Vimizim for MPS IVA

Yesterday was a historic day for individuals with MPS IVA, and the National MPS Society played a huge part!  The FDA Advisory Committee met in Washington, DC to consider approving Vimizim (elosulfase alfa), the enzyme replacement therapy (ERT) for MPS IVA (Morquio syndrome).  And they voted YES!

The Society has been involved with the Vimizim clinical trials from the beginning.  Barbara Wedehase, the Society’s Executive Director, attended the initial investigators’ meeting in June 2011 where information about the phase III clinical trial was presented in detail by BioMarin Pharmaceutical, the sponsor of the clinical trials.  Both Barbara and Laurie Turner, Program Director, helped with patient recruitment for the clinical trials, shared information and results about the clinical trials, and participated in a variety of patient advisory meetings.

For the last few months Barbara has been working closely with BioMarin to prepare for the FDA Advisory Committee meeting and with NORD to coordinate travel arrangements.  We know from our experience in 2003, with the Aldurazyme (ERT for MPS I) FDA Advisory Committee meeting, that the presentations from the families were the critical tipping point for approval.  A lot was at stake for Vimizim.

Barbara contacted six families, all of whom had a different story to tell, and worked with them to prepare for the big day.  We met Monday in Washington, DC where everyone practiced their presentation, then drove together to the FDA at 7am yesterday.  We were in a high state of nervous excitement, and after BioMarin and the FDA presented in the morning, it was our turn.

The families were beyond amazing. Their finely-tuned, five minute talks brimmed with heartfelt details about the devastating disease they face daily.  After hearing the statistics and data from BioMarin and the FDA, the Committee heard the true impact of Vimizim – greater stamina – to learn, socialize, engage; decreased pain; increased independence in daily living; and stopping the disease progression.  The committee members blotted away their tears while listening intently.  And they heard.

The committee voted 20 YES to approve and 1 to not approve.  Now the ball is in the FDA’s court to make the final decision, expected by February 28, 2014.

The Society thanks and congratulates the following individuals who spoke and their families who joined them at the microphone:  Maurizo Bellassai, Stephanie Bozarth, Mary Cavanagh, Nick Gaudin, Doug Kreul, and Sarah Van Orden.   Five of the speakers represented the many heroes who participated in the MPS IVA ERT clinical trials, and Nick represented the rest of the MPS IVA population who are waiting for approval to be treated. Diane Dorman from NORD and Steve Holland, the National MPS Society’s President, rounded out the public speakers providing testimony on their respective organization’s desire to see Vimizim approved. It was truly a team effort that made history yesterday!

As Steve Holland eloquently stated yesterday to the FDA, “the Society will NOT rest until there are treatments and cures for ALL the MPS and related diseases.”  Our fight for you continues.

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