US gene therapy company Apic Bio said that APB-102, its ALS SOD1 gene therapy,  has been granted orphan drug designation by the US Food and Drug Administration (FDA).

APB-102 is expected to shortly enter into clinical development for the genetic disorder – SOD1 amyotrophic lateral sclerosis (ALS).

The orphan designation is given to novel drugs that are targeted for the treatment of rare diseases that affect less than 200,000 people in the US.

Commenting on the FDA orphan designation for ALS SOD1 gene therapy APB-102, John Reilly – CEO and Co-Founder of Apic Bio said: “This orphan drug designation represents an important recognition by the FDA of APB-102’s potential to treat SOD1 ALS, a rare genetic form of ALS.

“Current treatments only offer modest benefits and do not target the genetic cause of the disease, leaving a significant unmet need.”

The orphan designation gives sponsors with development and commercial incentives, which include seven years of market exclusivity in the US, consultation by FDA on clinical study design, possibility for expedited drug development, and some fee exemptions and reductions.

ALS is estimated to affect 1.5 to 2.5 people in 100,000 in the US. It is estimated that 10% of all cases are thought to be inherited as a dominant trait, a condition called Familial ALS (FALS.) Nearly 15-20% of FALS cases are due to mutations in the gene that generates the copper zinc superoxide dismutase 1 (SOD1) enzyme, which results in a progressive degeneration of motor neurons hampering movement and muscle control.

Robert Brown – Professor of Neurology at the University of Massachusetts Medical School and scientific co-founder of Apic Bio, commenting on the FDA orphan designation for ALS SOD1 gene therapy APB-102, said: “It is gratifying that a clinical trial is being planned for this serious neurological disorder caused by SOD1 mutations; it has been 30 years since this mutation was first identified and now is the time to move toward therapy.”

For more updates on the ALS SOD1 gene therapy APB-102 and other pharma regulatory news, keep following Pharma News Daily.

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