Rare disease specialist Ultragenyx has received accelerated approval from the FDA for its gene therapy Genglycos, which treats glycogen storage disease type Ia (GSDIa), a rare metabolic condition. This marks the fifth FDA approval for the California-based biotech.
GSDIa is an ultra-rare condition in the United States, occurring in approximately 1 in 125,000 births. Estimates suggest around 1,500 to 2,500 people are living with the condition, although the precise figure is uncertain.
Genglycos is a one-time intravenous adeno-associated viral vector (AAV) gene therapy designed to deliver a functional G6PC gene to liver cells. The goal is to renew production of glucose-6-phosphatase in the liver, enabling more normal glucose release from glycogen stores.
The therapy is intended for children aged 8 years and above, as well as adults. It aims to reduce reliance on the current treatment of a strictly controlled diet and frequent uncooked-cornstarch doses.
The approval was based on results from a 48-week Phase III trial with 44 participants, which showed a statistically significant 31% mean reduction in daily cornstarch requirements versus placebo. The study demonstrated a meaningful reduction in treatment burden.
Related: Molecular Switch Found Behind Triple-Negative Cancer Spread
However, it did not establish improved long-term clinical outcomes, such as fewer hypoglycemic events. Confirmatory studies will be required to verify long-term clinical benefit.
Ultragenyx has agreed to provide safety and efficacy clinical data from open-label treatment of 50 patients and 20 control patients for two years.
There were seven serious adverse reactions in the study, including two anaphylaxis or infusion reactions, two adrenal-gland events, two cases of lactate levels that were higher than normal, and one hypoglycemia event. Like many AAV therapies, Genglycos comes with a risk of raised liver enzymes.
Some of the adverse events were linked to complications associated with the corticosteroids used to manage post-infusion liver inflammation.
David Weinstein, MD, one of the world’s leading GSDIa experts, states that day-to-day management of GSDIa requires a relentless regimen of raw cornstarch and strict dietary management, which can be extraordinarily demanding for patients and families.
Related: Einstein Mistake Redefines Universe Expansion Theory
The approval of Genglycos represents a major step forward for the GSDIa community, reflecting almost 30 years of work and scientific progress aimed at improving safety and the quality of life of people living with this disease.
No mention of treatment price was made in the company’s announcement, but Ultragenyx will provide support to help enrolled patients and caregivers access treatment through its UltraCare program.
The FDA approval of Genglycos is a significant development in the treatment of GSDIa, and research on glycogen storage diseases will continue to be important for understanding the efficacy and safety of therapies like Genglycos.
They will continue to monitor the situation.
Leave a Reply