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Focalin XR(TM) Clinical Studies Suggest Investigational Once-Daily ADHD Treatment is Safe and Effective in Both Children and Adults

Posted on: Monday, 25 October 2004, 08:00 CDT

WARREN, N.J., Oct. 25 /PRNewswire-FirstCall/ -- Celgene Corporation announced that its investigational drug Focalin XR (formerly known as Focalin LA) may help adults and children manage Attention- Deficit/Hyperactivity Disorder (ADHD) symptoms, according to three separate studies presented at the 51st Annual Meeting of the American Academy of Child and Adolescent Psychiatry (AACAP). Results from one of the pediatric studies show that Focalin XR (dexmethylphenidate HCl) extended release capsules may help treat ADHD symptoms for 12 hours. ADHD is one of the most common psychiatric disorders of childhood and is estimated to affect five to seven percent of children and approximately four percent of the adult population.

The Division of Neuropharmacological Drug Products at the U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) submitted by Novartis, seeking approval to market Focalin XR for the treatment of Pediatric and Adult ADHD. The Focalin XR NDA submitted by Novartis Pharma AG triggered a $7.5 million milestone payment.

Adult Study

The adult study was designed to evaluate the long-term efficacy, safety and tolerability of Focalin XR 20 mg to 40 mg daily. The study was a 5-week, multicenter, double blind, randomized, placebo-controlled study followed by a 6-month open-label extension. Participants included 221 men and women aged 18 to 60 years who completed the 5-week double blind clinical trial and 170 patients who continued into the 6-month open-label extension phase. All subjects had a diagnosis of ADHD and the average age was 38.7 years. In the double-blind study, the primary efficacy analysis compared the two higher doses of Focalin XR (30 and 40 mg) versus placebo. The secondary efficacy analysis compared the effects of Focalin XR 20 mg versus placebo. Efficacy was measured using the Diagnostic and Statistical Manual-IV (DSM-IV) ADHD Rating Scale, a standard assessment tool used in ADHD. Secondary measures included evaluations rated by patients, clinicians and observers.

At the end of the double-blind study, Focalin XR was statistically superior to placebo on both primary comparisons. The mean change from baseline on the rating scale total score was significantly greater with all doses of Focalin XR than with placebo. Score changes were 7.6 for placebo, 13.3 for patients taking the 20 mg dose of Focalin XR (P=.006), 12.9 for patients treated with the 30 mg dose (P=.012) and 16.5 (P<.001) for patients taking the 40 mg dose (all P values vs. placebo).

At the end of the open label extension, treatment response was evaluated in 103 patients. Patients who switched from placebo to Focalin XR (n=20) showed significant clinical improvement and patients continuing treatment with Focalin XR (n=82) continued to show increasing benefits over six months, based on changes in the DSM-IV ADHD Rating Scale.

Focalin XR was generally well tolerated. The most frequently reported adverse events included headache, insomnia, and decreased appetite.

Pediatric Studies

Data presented at AACAP suggest that Focalin XR is safe and effective in the treatment of children and adolescents with ADHD. One hundred three ADHD patients aged 6 to 17 years participated in a double blind, placebo- controlled, parallel group study to compare the efficacy and safety of Focalin XR to placebo. Efficacy was evaluated by examining the presence of ADHD symptoms in school, home and clinical environments. In the study, the primary outcome was measured by evaluating change from baseline using the Conners' ADHD/DSM-IV Scales for Teachers (CADS-T). Secondary efficacy measures included evaluations rated by teachers, parents and clinicians. At the end of the study, the mean change from baseline on the CADS-T total subscale score was 16.3 for Focalin XR and 5.9 for placebo (P<.001). Outcomes on all secondary efficacy measures were also statistically significant in favor of Focalin XR.

In a second pediatric study also presented today at AACAP, investigators evaluated the efficacy of Focalin XR 20 mg/day at various points throughout the day up to 12 hours. The randomized, double-blind, crossover study included 54 children aged 6 to 12 years with a DSM-IV diagnosis of ADHD who were previously stabilized on methylphenidate 20 to 40 mg/day. Investigators assessed improvement in attention and behavior among study participants in a classroom setting at specific intervals for up to 12 hours post-dose. Improvements were measured using the Swanson, Kotkin, Agler, Mylnn, and Pelham (SKAMP) rating scale, a standard assessment tool used in clinical trials to evaluate attention and behavior. Focalin XR was found to be significantly more effective than placebo in treating ADHD symptoms, as measured by the SKAMP rating scale, at each time point measured, including 12 hour time point.

In both pediatric studies, Focalin XR was generally well tolerated. Adverse events were consistent with those observed in other studies with methylphenidate and dexmethylphenidate.

About ADHD

ADHD is a neurobiologic disorder that interferes with an individual's ability to regulate activity level and behavior, and sustain focus in developmentally appropriate ways. In adults, it is associated with poor functioning in educational and occupational environments, as well as emotional, social and economic problems.

In children and adults, the core symptoms of ADHD are inattention, impulsivity and hyperactivity; however, in adults symptoms of hyperactivity often subside with maturity or may manifest differently. ADHD is one of the most common psychiatric disorders of childhood and is estimated to affect five to seven percent of children. Many children with ADHD will continue to experience symptoms into adulthood. ADHD is estimated to affect approximately four percent of the adult population.

About Focalin(TM) and Focalin(TM) XR

Focalin(TM) (dexmethlyphenidate hydrochloride) containing only the effective d-isomer, is a refined form of Ritalin(R) (d,l-thero-methlyphenidate hydrochloride), providing favorable tolerability and dosing flexibility at only half the dose. Both Focalin and Ritalin LA have been approved by the FDA for the treatment of ADD/ADHD in children and adolescents. Focalin XR, an extended release version, is intended to reproduce twice-daily dosing of Focalin immediate release tablets. Focalin XR NDA was accepted by FDA for review as treatment for pediatric and adult ADHD indications.

Celgene licensed the worldwide rights (excluding Canada) to Focalin and Focalin XR to Novartis Pharma AG in exchange for milestone payments and royalties on Focalin and the entire Ritalin family of drugs including Ritalin, Ritalin LA, Ritalin SR and ultimately Focalin XR. Celgene retained the rights to Focalin for oncology-related indications including chemotherapy-induced cognitive dysfunction and cancer fatigue and is currently evaluating Focalin for this indication.

About Celgene

Celgene Corporation, headquartered in Warren, New Jersey, is an integrated biopharmaceutical company engaged primarily in the discovery, development and commercialization of novel therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit the Company's website at http://www.celgene.com/.

This release contains certain forward-looking statements which involve known and unknown risks, delays, uncertainties and other factors not under the Company's control, which may cause actual results, performance or achievements of the Company to be materially different from the results, performance or other expectations implied by these forward-looking statements. These factors include results of current or pending research and development activities, actions by the FDA and other regulatory authorities, and those factors detailed in the Company's filings with the Securities and Exchange Commission such as 10K, 10Q and 8K reports.

Celgene Corporation

CONTACT: Robert J. Hugin, Senior VP and CFO, +1-732-271-4102, or BrianP. Gill, Director PR-IR, +1-732-652-4530, both of Celgene Corporation

Web site: http://www.celgene.com/

Source: PRNewswire-FirstCall

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