FDA Awards Rare Pediatric Disease and Orphan Drug Designations to BGTC Programs

Rare Daily Staff

The U.S. Food and Drug Administration has granted the National Institutes of Health’s National Center for the Advancing of Translational Sciences both Rare Pediatric Disease designations and Orphan Drug Designations for five experimental gene therapies in development through its Accelerating Medicines Partnership Bespoke Gene Therapy Consortium.

NCATS said the designations are key to the Bespoke Gene Therapy Consortium’s (BGTC) mission to streamline the development of bespoke gene therapies for extremely rare diseases where no commercial interest exists.

“Getting these designations increases the chance that the successful therapies will be picked up by a company and commercialized,” said NCATS Director Joni Rutter,

BGTC, a public-private partnership co-led by NCATS, aims to speed the creation of gene therapies for rare diseases. The new designations are expected to make the BGTC gene therapies more attractive to companies for development.

The FDA grants a Rare Pediatric Disease designation when preclinical data for an experimental drug or therapy suggest the treatment could be effective in treating a rare disease that primarily affects children. Similarly, the FDA grants an Orphan Drug designation for an experimental treatment when preclinical data show a possible benefit for people with a rare disease. Both designations can open a door to financial incentives for prospective partners to further develop the therapies for rare diseases and bring them to market.

The Rare Pediatric Disease designation can enable the award of a Priority Review Voucher if a gene therapy is approved. A company that receives an approval for a drug for a rare pediatric disease can use the PRV to speed the FDA review process for another product. The voucher is potentially lucrative because it is transferable. Many holders have sold their vouchers to other drug developers for around $100 million each.

Incentives from the Orphan Drug designation include an exemption from the user fee for the FDA New Drug Application or the Biologics License Application. This fee can be millions of dollars. Companies can get tax credits for successful clinical trials with their drug products. Recipients also get seven years of exclusively to market an approved drug.

The five BCGT gene therapies include experimental ones for the metabolic disease propionic acidemia, the progressive brain disorder multiple sulfatase deficiency, the degenerative eye disease NPHPR mutation-associated retinal dystrophy, the inherited eye disease autosomal recessive retinitis pigmentosa due to CNGB1 mutation, and the lysosomal storage disorder mucopolysaccharidosis IVA (Morquio A syndrome).

A Rare Pediatric Disease designation and an Orphan Disease designation also were granted for a sixth program within the BGTC for a gene therapy to treat the genetic eye disorder congenital hereditary endothelial dystrophy. The sponsor of this program is Anthony Aldave at the University of California, Los Angeles.

Photo: Joni Rutter, Director of NCATS