Business Honor: Ultragenyx Gene Therapy Wins FDA Approval

Ultragenyx Gene Therapy Wins First FDA Approval for Rare Metabolic Disorder

Life Sciences and Bio Pharma

Ultragenyx’s Genglycos has received the FDA’s first approval for a gene therapy targeting glycogen storage disease type Ia, a rare metabolic disorder.

The gene therapy developed by Ultragenyx, known as Genglycos, has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of glycogen storage disease type Ia (GSDIa). This is the first time the FDA has approved a gene therapy to treat the rare metabolic disorder.

GSDIa is also referred to as Von Gierke disease. It is an ultra-rare genetic disorder due to the lack of an enzyme required to release glucose from the liver into the bloodstream. According to Ultragenyx, the number of people living with this disorder in the US ranges from 1,500 to 2,500.

This condition leads to dangerous bouts of low blood sugar since the liver cannot control glucose level. The patients have to take raw cornstarch orally to replace glucose.

Genglycos is developed to help alleviate the root cause of GSDIa by facilitating the process of glycogen degradation to glucose. The drug can decrease the need for cornstarch among patients.

Ultragenyx disclosed that the drug’s list price in the United States is projected to be $2.7 million per patient, and it should be available to patients in about 30 to 60 days after qualification at designated treatment centers.

The FDA’s approval was based on a 48-week late-stage clinical trial where patients who were administered Genglycos showed a decreased need for cornstarch compared to patients under placebo. Nevertheless, the decreased cornstarch need is a surrogate endpoint rather than a clinical benefit.

The FDA has thus mandated additional clinical trials to prove the efficacy of the drug. According to Ultragenyx, it has agreed to furnish additional data on safety and efficacy for two years after treatment.

The approval comes amid the efforts of other firms to develop a cure for GSDIa. For instance, Moderna and Beam Therapeutics are working on experimental drugs, though they are still in the initial stages of clinical trials. Business Honor believes the approval highlights the potential of gene therapy to address the underlying causes of ultra-rare diseases while underscoring the need for continued clinical evidence.