|Shire's Vyvanse Approved in US; Raptor's PROCYSBI Receives FDA Approval|
|By Staff and Wire Reports|
|Wednesday, 01 May 2013 19:31|
Shire plc (NASDAQ: SHPG), the global specialty biopharmaceutical company, announced the US FDA approved the prescription medication Vyvanse® (lisdexamfetamine dimesylate) Capsules, (CII) as a maintenance treatment in children and adolescents with Attention-Deficit/Hyperactivity Disorder (ADHD). Vyvanse is currently approved as a maintenance treatment in adults with ADHD. With this new approval, Vyvanse becomes the only stimulant approved for maintenance treatment in children, adolescents, and adults (patients ages 6 and above) with ADHD.
The approval is based on results from a 32-week study: 26 weeks of open-label treatment with Vyvanse followed by a 6-week randomized withdrawal phase. The study was designed to evaluate the continued efficacy of Vyvanse in children and adolescents (aged 6 to 17 years). A significantly lower proportion of treatment failures occurred among Vyvanse patients (15.8%) compared to placebo (67.5%) at end point of the randomized withdrawal period, showing that significantly more patients treated with Vyvanse maintained ADHD symptom control compared with placebo.
Vyvanse is a Schedule II controlled substance. CNS Stimulants (amphetamines and methylphenidate-containing products) have a high potential for abuse and dependence. Assess the risk of abuse prior to prescribing and monitor for signs of abuse and dependence.
To evaluate the efficacy of Vyvanse for maintenance treatment in children and adolescents with ADHD, Shire elected to conduct a double-blind, placebo-controlled, randomized withdrawal clinical trial. In this design, patients who respond to a treatment are randomized to continue receiving that treatment or placebo. Using the proportion of patients experiencing symptom relapse as a primary outcome, this type of study in patients with ADHD can be used to demonstrate long-term efficacy in lieu of conducting a long-term, placebo-controlled, parallel-group study. The utility of this design is that the period of placebo exposure, with the potential for worsening of ADHD symptoms, is relatively short.
The double-blind, placebo-controlled, randomized withdrawal study was conducted in 276 children and adolescents aged 6 to 17 with ADHD. Of these patients, 236 participated in a preceding study and 40 directly enrolled. The study consisted of 4 phases:
Raptor Pharmaceutical (Nasdaq: RPTP) announced the U.S. FDA has approved PROCYSBI™ (cysteamine bitartrate) delayed release capsules for the treatment of nephropathic cystinosis in adults and children 6 years and older. In a Phase 3 study, PROCYSBI showed consistent cystine depletion over the full 12-hour dosing period. Sustained levels of cysteamine have not historically been achieved in the majority of patients in this population. Studies have shown that sustained cystine depletion in patients may significantly delay disease progression, including kidney dysfunction, dialysis, kidney transplant, organ failure and premature death.
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"PROCYSBI's approval advances Raptor closer to its goal of becoming an integrated, commercial-stage biopharmaceutical company. It is especially gratifying for us that patients and caregivers in the cystinosis community now have a long-anticipated treatment available to them," said Christopher M. Starr, Ph.D., CEO and co-founder of Raptor. "PROCYSBI is the result of a decade-long patient- and physician-initiated effort to improve the treatment and lives of cystinosis patients. We are very grateful to have worked together on the development, manufacturing and regulatory submission of this important treatment for the cystinosis community."
PROCYSBI is the culmination of early research funded by the patient advocacy group Cystinosis Research Foundation. The FDA approval of PROCYSBI was based on a New Drug Application (NDA) comprising data from six clinical trials, including a multi-center randomized, active-controlled Phase 3 trial of 43 patients with nephropathic cystinosis and extension data from that trial. The most commonly reported adverse reactions were vomiting, abdominal pain/discomfort, headaches, nausea, diarrhea, anorexia/decreased appetite, breath odor, fatigue, dizziness, skin odor and rash. Clinical studies are ongoing in children less than six years of age to evaluate for the first time the safety and efficacy of PROCYSBI. Over ninety percent of the cystinosis population is six years or older.
"Sustaining appropriate levels of cysteamine in the body is the key to maintaining organ function and lowering the likelihood of kidney transplantation. Most patients don't take their cystinosis medication consistently as a result of severe stomach side effects and a burdensome dosing schedule," said Craig Langman, M.D., The Isaac A. Abt, MD Professor of Kidney Diseases Feinberg School of Medicine and Head of Kidney Diseases at Ann & Robert H. Lurie Children's Hospital of Chicago. "In addition to providing sustained control of cystine levels, PROCYSBI's dosing schedule and side effect profile may help patients stay the course with their treatment. The fact that 40 of 41 patients from the Phase 3 trial elected to enroll in the extension study and have been followed for two years now demonstrates their motivation to be on PROCYSBI therapy."
Physicians can now prescribe PROCYSBI by calling RaptorCares at 1-855-888-4004. RaptorCares provides individualized services to help patients access PROCYSBI through education, support, extensive case management and a commitment to the principle that no eligible U.S. patient with nephropathic cystinosis will be denied access to PROCYSBI based on inability to pay. Raptor intends to begin shipping PROCYSBI within 6-8 weeks.
The FDA approval of PROCYSBI triggers the second of two $25 million payments to Raptor under the terms of the HealthCare Royalty Partners (HC Royalty) $50 million loan agreement. The loans mature on December 31, 2019, bear interest at an annual fixed rate of 10.75%, and a Synthetic Royalty® variable rate, tiered down, based on a percentage of future PROCYSBI sales. Raptor signed the loan agreement with HC Royalty in December 2012 and received the first $25 million payment upon closing. The loan is interest-only for the first two years.
Conference Call Raptor has scheduled an investor conference call and webcast to discuss the FDA approval of PROCYSBI at 4:30 p.m. EDT (1:30 p.m. PDT) today, April 30, 2013. Individuals interested in participating in the call may do so by dialing 888-539-3678 for domestic callers or 719-325-2315 for international callers. A telephone replay will be available for 48 hours following the conclusion of the call by dialing 888-203-1112 for domestic callers, or 719-457-0820 for international callers, and entering reservation code 3352728. The live conference call will also be available in the Investor Relations section of the company's website at www.raptorpharma.com. A recording of the call will be available on the company's website for 14 days following the completion of the call.
About Nephropathic Cystinosis Nephropathic cystinosis comprises 95% of cystinosis, a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body. Elevated cystine leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death. Nephropathic cystinosis is usually diagnosed in infancy and requires lifelong therapy. Left untreated, the disease is usually fatal by the end of the first decade of life. There are an estimated 500 patients living in the U.S. with cystinosis, and 2,000 worldwide.
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