Home > Media > Details
FDA Grants Rare Pediatric Disease Designation (RPDD) to Angitia's AGA2115 for the Treatment of Osteogenesis Imperfecta

Angitia is delighted to announce that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to Angitia's investigational therapy AGA2115 for the treatment of Osteogenesis Imperfecta (OI), a life-threatening genetic skeletal disease, under Section 529(a)(3) of the Federal Food, Drug, and Cometic Act (the FD&C Act) (21 U.S.C. 360ff(a)(3)).

AGA2115 is a first-in-class investigational drug candidate discovered by Angitia, for the treatment of OI. Data from preclinical disease models showed a robust effect of AGA2115 in restoring bone mass and bone strength, improving bone quality and reducing skeletal fractures. These results demonstrated the potentials of AGA2115 for the treatment of OI.

About OI:

Osteogenesis imperfecta (OI) is an inherited connective tissue disorder with pathophysiology driven by abnormal collagen metabolism resulting in skeletal deformity, bone fragility, reduced bone mass, and variable extra-skeletal symptoms. As a rare pediatric disease, OI presents as a spectrum ranging from mild to severe and life-threatening. Disease manifestations occur in the neonatal and pediatric age groups, presenting with frequent and recurrent fractures, often elicited by little or no trauma. Severe OI cases manifest with multiple debilitating fractures resulting in loss of independent ambulation, deformity, and stunted growth, with the most severe cases presenting with perinatal lethality. Associated features may include, but are not limited to, muscle weakness, joint laxity, issues with dentition, hearing loss, and skeletal malformations.

About RPDD:

The RPDD program is intended to encourage development of new drug and biological products (the “drug”) for the prevention and treatment of certain rare pediatric diseases. FDA will designate a drug as a drug for a “rare pediatric disease” if the sponsor provides adequate information to demonstrate that the drug is for a rare pediatric disease (a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents) and an adequate explanation, with supportive data, of the drug’s mechanism of action suggesting that the drug may be effective in the rare pediatric disease. And if the biologics license application (BLA) of AGA2115 is approved by FDA, Angitia will be eligible to receive a priority review voucher, which can be used to reduce the FDA review time on a new drug application to six months from ten months. The vouchers are also transferrable which entitle the bearer to priority review for a future new drug application.