Repligen Corporation announced that the U.S. FDA has granted Fast Track designation for secretin for the treatment of pediatric autism.
“We are very pleased that the FDA has designated this program for Fast Track status,” stated Walter C. Herlihy, President and CEO of Repligen Corporation.
“We believe that this action affirms a heightened awareness to the great need for therapeutic interventions for autism and we look forward to continued positive interactions with the FDA.” In April of this year, Repligen completed a Phase 2 clinical trial which evaluated three doses of secretin or a placebo in 126 children, 3 to 6 years of age, with moderate to severe symptoms of autism and reported gastrointestinal symptoms.
A stool sample was collected from each patient at the beginning of the trial and subsequently analyzed for two proteins: calprotectin, a marker of gastrointestinal inflammation or colitis and chymotrypsin, an enzyme released from the pancreas during digestion.
For each protein, 25-30% of the patients had an abnormal value prior to treatment with secretin or a placebo.
A statistically significant treatment effect of secretin was demonstrated in the 64 patients, or 51% of the total patient population, with normal levels of both proteins.
In this subgroup, there was a statistically significant effect of secretin by four endpoints including improvement in social function as determined with the Autism Diagnostic Observation Schedule (p=0.02), overall symptom changes as determined by the Clinical Global Impression Scale by both a professional (p=0.01) and independently a parent (p<0.001) and an increase in receptive language as determined by the MacArthur Communicative Development Inventory (p=0.02). These data have been communicated to the FDA as the basis for a discussion of the next step in the development of secretin for autism.
Repligen Corporation develops new drugs for debilitating pediatric disorders including autism, cancer, and immune and metabolic disorders.