FDA News

FDA grants orphan drug, rare pediatric disease status to gene therapies for Sanfilippo syndrome

The FDA has granted both orphan drug designation and rare pediatric disease designation to ABX-101 and ABX-102, two gene therapies for the treatment of Sanfilippo syndrome type A and B, according to the drug’s manufacturer.

The two products (PlasmaTech Biopharmaceuticals, Inc.), are gene-therapy programs intended to treat Sanfilippo syndromes type A and B (MPS IIIA and MPS IIIB), two of the four deadly genetic diseases resulting from the body’s inability to properly break down certain sugars.

“We are very pleased to announce that the FDA has provided these two important designations to our Sanfilippo drug candidates, reflecting their potential to address the significant unmet medical needs of children with these rare and devastating lysosomal storage diseases,” Tim Miller, PhD, president and CEO of PlasmaTech Biopharmaceuticals said in a press release.

Unlike many other forms of mucopolysaccharidoses (MPS), the symptoms of Sanfilippo syndrome manifest after the first year of life, exhibited by developmental/behavioral difficulties followed by progressive muscular and cognitive decline after the age of two.

The FDA based its decision on preclinical studies that demonstrated the therapies were well-tolerated with minimal side effects. In addition, efficacy studies have exhibited extended therapeutic benefit months following treatment.

“These important FDA designations reflect the critical nature of these two programs to these patients and families,” Steven H. Rouhandeh, executive chairman of PlasmaTech said in the release. “PlasmaTech’s programs in MPS IIIA and IIIB address compelling unmet medical needs with breakthrough technology. We are committed to building on these achievements, and intend to continue to expand our product pipeline in the rare disease space.”

PlasmaTech has partnered with patient advocate groups, researchers and clinicians, to tentatively begin clinical trials for ABX-101 and ABX-102 in 2015.

The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

The FDA has granted both orphan drug designation and rare pediatric disease designation to ABX-101 and ABX-102, two gene therapies for the treatment of Sanfilippo syndrome type A and B, according to the drug’s manufacturer.

The two products (PlasmaTech Biopharmaceuticals, Inc.), are gene-therapy programs intended to treat Sanfilippo syndromes type A and B (MPS IIIA and MPS IIIB), two of the four deadly genetic diseases resulting from the body’s inability to properly break down certain sugars.

“We are very pleased to announce that the FDA has provided these two important designations to our Sanfilippo drug candidates, reflecting their potential to address the significant unmet medical needs of children with these rare and devastating lysosomal storage diseases,” Tim Miller, PhD, president and CEO of PlasmaTech Biopharmaceuticals said in a press release.

Unlike many other forms of mucopolysaccharidoses (MPS), the symptoms of Sanfilippo syndrome manifest after the first year of life, exhibited by developmental/behavioral difficulties followed by progressive muscular and cognitive decline after the age of two.

The FDA based its decision on preclinical studies that demonstrated the therapies were well-tolerated with minimal side effects. In addition, efficacy studies have exhibited extended therapeutic benefit months following treatment.

“These important FDA designations reflect the critical nature of these two programs to these patients and families,” Steven H. Rouhandeh, executive chairman of PlasmaTech said in the release. “PlasmaTech’s programs in MPS IIIA and IIIB address compelling unmet medical needs with breakthrough technology. We are committed to building on these achievements, and intend to continue to expand our product pipeline in the rare disease space.”

PlasmaTech has partnered with patient advocate groups, researchers and clinicians, to tentatively begin clinical trials for ABX-101 and ABX-102 in 2015.

The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.