On July 24, 2006, the FDA approved Elaprase. It provided HOPE.
It makes me wonder what I was doing at the time, since it wasn’t very long ago.
Interestingly, it was probably about that time that I discovered I was pregnant with Case. As his body grew, all the while replicating cells that were defective, lacking an enzyme to break down glycosaminoglycans, others were celebrating a drug that would become a central part of our everyday lives.
While other moms were fighting for access to this drug, one being my eventual close friend, Deb Purcell, I was still living in the innocence of believing that children are healthy, that the chubby lips and cheeks were uniquely him alone, and that the episodes of him stopping breathing would subside on their own.
I had no knowledge of Elaprase, Shire, the Orphan Drug Act, or its key role in encouraging the development of drugs like Elaprase. Almost 3 years after the approval of Elaprase, I would discover just how important that event was.
Were it not for the parents and children who participated in the original natural history study and the original Elaprase studies, I have no doubt that we would not be where we are today, on the cusp of transforming the entire diagnosis, management, and prognosis for the disease.
I no longer want parents to have to hear the words, “Your child is going to die.”
This year was also the 30th anniversary of the passing of the Orphan Drug Act, the legislation that encourages companies to develop orphan drugs by granting them 7 years of market exclusivity, tax breaks, and other benefits. Although it’s been 30 years since the passing of the Orphan Drug Act, I think we’ve come a long way during that time.
And now that the 7-year market exclusivity of Elaprase as a treatment for Hunter Syndrome has expired, we should see competition enter the market. We’ve already seen that Green Cross will be applying for an IND (investigative new drug) for their Hunterase drug as a biobetter of Elaprase. In addition, Armagen had just received an orphan drug designation for its AGT-182 compound and expects to enter clinical trials in 2014. We understand this to be a drug to treat the brain disease, which would be fantastic for all those who have not been able to access the Shire IT drug.
Happy birthday Elaprase!