Conclusions

The development of a treatment for a rare disorder like MPS I is challenging and rewarding for the drug development team. Aldurazyme is the first ERT for an MPS disorder and represents a major step forward for patients with these rare disorders. The biology and science of enzyme production, uptake, and clinical development is unique in the lysosomal and MPS disorders. The challenges of using surrogate endpoints and the design of studies in heterogeneous populations are most difficult in MPS I precisely because it is so rare. There is also no greater thrill in drug development than the approval of a safe and effective drug that will change the practice of medicine for a rare, untreatable disease like MPS I. The approval of this product represents the combined efforts of scientists, drug development team, regulators, and patients who persistently pursued a solution to a most devastating disease.

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