This trial will be the first under Autism Speaks' Clinical Trials Network (CTN):
Neuropharm Group plc (AIM: NPH), a speciality pharmaceutical company focused on neurodevelopmental disorders, is pleased to announce that the US Food and Drug Administration (FDA) has granted Fast Track designation for Neuropharm’s program to develop NPL-2008 in the treatment of Autism Disorder.
NPL-2008, a new orally disintegrating tablet of low-dose fluoxetine, is currently in Phase III development for the treatment of the core symptom of repetitive behaviours in patients five years of age or older with Autistic Disorder.
On its website, www.fda.gov, FDA states: “Because fast track products are intended to treat serious or life-threatening conditions and must demonstrate the potential to address unmet medical needs for such conditions, an NDA for a product in a fast track development program ordinarily will be eligible for priority review and FDA may consider for review, portions of a marketing application before the complete NDA is submitted. Fast Track is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously.”
The NPL-2008 program received Orphan Drug Designation from FDA in 1999, giving the product the potential for seven years of market exclusivity on receipt of regulatory approval. In addition the FDA Office of Orphan Drug funded a clinical study in children and adolescents at Mount Sinai School of Medicine in New York followed by a second study in adults.
Neuropharm’s current Phase lll study (SOFIA) underwent Special Protocol Assessment by FDA in 2007. The study is currently ongoing at 17 sites in the US in collaboration with Autism Speaks, the US advocacy organization, and its Clinical Trials Network of expert investigators. The results from this randomised, double-blind trial in more than 120 child and adolescent patients with Autistic Disorder will form part of the NPL-2008 New Drug Application, which is targeted for submission to FDA in Q4 2008.
Robert Mansfield, Neuropharm’s CEO, commented: “We are delighted to announce that FDA has granted Fast Track designation for our program. The point was made earlier this month on World Autism Awareness Day that more children will be diagnosed with autism this year than with diabetes, cancer and AIDS combined. Fast Track designation emphasises the importance we believe should be accorded to this condition.”
SOFIA is the first industry-sponsored trial for Autism Speaks' Clinical Trials Network (CTN), and will include over 120 children and adolescents with autism enrolled across the U.S. at participating CTN sites. The study is evaluating the efficacy of Neuropharm's new melt-in-the-mouth formulation for the treatment of repetitive behaviors in autism. This study will be a crucial part of an application to receive an autism indication for this formulation of fluoxetine.
I have no doubt that for those parents whose children are presently taking Prozac or who are currently considering it, such a trial will be a relief. With the majority of medications, trials are typically done on adults. The effects of the drugs on those under 18 are often unknown (and, especially unknown when it comes to individuals with autism). Bad reactions in the pediatric community only come to light once a serious side-effect has occurred.
Prozac was approved for pediatric use in 2003 and is generally well-tolerated. A trial focused on a pediatric autistic population (for both efficacy and side-effects) will benefit many. A dissolving tablet will be much easier for children to tolerate, as well.