Rare Disease Report

PBI-4050 Receives Rare Pediatric Disease Designation for Treatment of Alström Syndrome

AUGUST 07, 2018
Krista Rossi
This morning, the US Food and Drug Administration (FDA) has granted a rare pediatric disease designation to Prometic Life Sciences Inc.’s PBI-4050 for the treatment of patients with Alström syndrome.

"This is the first pediatric designation granted by the FDA to our small molecule drug candidate PBI-4050 and the third overall, following the previous 2 granted for our plasma-derived therapeutics candidates,” said Mr. Pierre Laurin, president and CEO of Prometic, in a recent statement. “This highlights the depth and value of our two drug discovery platforms. 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."

PBI-4050 is an orally active lead drug candidate with excellent safety and efficacy profiles demonstrated in a large number of animal models of fibrosis affecting different organs, including the lung, liver, heart, kidney, and pancreas.
 
A rare inherited autosomal recessive syndrome, 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,” according to a recent news release. A progressive loss of vision and hearing, a form of heart disease that weakens the heart muscle (dilated cardiomyopathy), and short stature are also characterizations of the rare disease that can cause serious or life-threatening medical issues.

PBI-4050 is now entering pivotal placebo-controlled phase 3 clinical trials for the treatment of idiopathic pulmonary fibrosis (IPF) and has already begun placebo-controlled phase 2 trials in metabolic syndrome and type 2 diabetes patients.

“Alstrom syndrome is considered a severe model for fibrotic conditions," said Pierre Laurin, CEO of Prometic, exclusively to Rare Disease Report®, "so with the success PBI-4050 has shown in the multi-organ fibrosis associated with this disease to date, we believe PBI-4050 has the potential to change the treatment paradigm for fibrosis.” 

Previously, PBI-4050 was granted an orphan drug designation by the FDA and the European Medical Agency (EMA) for the treatments of Alström syndrome and idiopathic pulmonary fibrosis (IPF). The treatment has also received a PIM (Promising Innovative Medicine) designation by the Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of IPF and Alström syndrome.

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