FDA Grants Rare Pediatric Disease Designation to Prometic’s Experimental Alsotröm Syndrome Drug

Rare Daily Staff

The U.S. Food and Drug Administration has granted Prometic Life Sciences a Rare Pediatric Disease designation for its small molecule drug candidate, PBI-4050, for the treatment of Alström syndrome, a rare and potentially life-threatening genetic disease.

It is the third Rare Pediatric Disease designation the FDA granted Prometic for its drugs in development.

Alström syndrome is characterized by the onset of obesity in childhood or adolescence, type 2 diabetes, often with severe insulin resistance, dyslipidemia, hypertension and severe multi-organ fibrosis involving the liver, kidney and heart. It can cause a progressive loss of vision and hearing, a form of heart disease that weakens the heart muscle, and short stature. The disorder can also cause serious or life-threatening medical problems involving the liver, kidneys, bladder, and lungs. The clinical manifestations of Alström syndrome vary in severity, and not all affected individuals have all of the features associated with the disorder.

Prometic’s PBI-4050 is an orally-active experimental drug that has demonstrated safety and efficacy in animal models of fibrosis affecting different organs, including the lung, liver, heart, kidney, and pancreas. The effects of PBI-4050 demonstrated in animal models have been replicated in phase 2 studies in idiopathic pulmonary fibrosis, in metabolic syndrome with type 2 diabetes, and in Alström syndrome. PBI-4050 is entering pivotal placebo-controlled phase 3 clinical trials for the treatment of IPF and has already begun placebo-controlled phase 2 trials in metabolic syndrome and type 2 diabetes patients.

The FDA grants Rare Pediatric Disease designations for serious or life-threatening diseases that primarily affect individuals aged from birth to 18 years. If approved, Prometic’s PBI-4050 could be eligible to receive a Rare Pediatric Disease Priority Review Voucher.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Ultragenyx and Kyowa Hakko Kirin sold their Priority Review Voucher for $80.6 million.

“This highlights the depth and value of our two drug discovery platforms,” said Pierre Laurin, president and CEO of Prometic. “We look forward to discussing the potential regulatory approval pathway to bring this innovative therapy to pediatric patients with Alström syndrome during our upcoming meeting with the FDA.”

In addition to the Rare Pediatric Disease Designation, PBI-4050 was previously granted Orphan Drug designation by the FDA and the European Medicines Agency for the treatments of Alström syndrome and idiopathic pulmonary fibrosis, as well as Promising Innovative Medicine designation by the Medicines and Healthcare products Regulatory Agency for the treatment of idiopathic pulmonary fibrosis and Alström syndrome.

August 7, 2018
Photo: Pierre Laurin, president and CEO of Prometic