We would like to express our sincere gratitude to Sanofi Genzyme for including New Zealand Pompe patients in their International Compassionate Access Program. Patients and families that have no other options for lifesaving treatments now have hope. – CAP

The press release from the New Zealand Pompe Network is below.

Masterton, New Zealand – (February 25, 2017) – The New Zealand Pompe Network is ecstatic to announce that Sanofi Genzyme has offered Pompe disease patients in New Zealand access to their International Compassionate Access Program (ICAP) for the drug Myozyme®.  A huge THANK YOU to Sanofi Genzyme for their generosity and their humanitarian program.

Pompe patients in New Zealand have been declined treatment by PHARMAC/Government for more than 10 years since Myozyme has been on the market.  However, Myozyme was approved for infantile patients in New Zealand from the 1st of December 2016.  We have no infantile patients in New Zealand, but we may get some at any time.  In January this year it was recommended that access again be declined for adults for this life saving drug.  This decision by the Pharmacology and Therapeutics Advisory Committee (PTAC) was devastating for the remaining 10 Pompe sufferers in New Zealand.  Sadly, one of our patients lost his battle with Pompe disease in December, while awaiting the decision from PTAC following their November 2016 meeting.

About Pompe Disease
Pompe disease is a rare, inherited and often fatal disorder that disables the heart and muscles.  It is caused by mutations in a gene that makes an enzyme called alpha-glucosidase (GAA).  Normally, the body uses GAA to break down glycogen, a stored form of sugar used for energy.  But in Pompe disease, mutations in the GAA gene reduce or completely eliminate this essential enzyme.  Excessive amounts of glycogen accumulate everywhere in the body, but the cells of the heart and skeletal muscles are the most seriously affected.  The disease commonly results in patients dying from respiratory failure after spending their remaining years wheelchair bound, on mechanical ventilation and being fed through a stomach tube.

About Myozyme
Myozyme (alglucosidase alfa) contains an enzyme that naturally occurs in the body in healthy people. Alglucosidase alfa helps replace this missing enzyme.  Myozyme has been the one and only treatment for the fatal Pompe disease since 2006.  This medication is available in 76 countries around the world.  Out of all of the OECD countries, only 3 refuse to fund Myozyme.  Iceland, Austria and New Zealand.

About the New Zealand Pompe Network
The New Zealand Pompe Network (NZPN) was started by Pompe disease patients in 2011. We are officially a non-profit society committed to supporting Pompe sufferers and their families.

Contacts
Allyson Lock
nz.pompe@gmail.com
nzpompenetwork.weebly.com

Source: New Zealand Pompe Network

Tagged: compassion, ERT