Shire's Vyvanse Approved in US; Raptor's PROCYSBI Receives FDA Approval Print E-mail
By Staff and Wire Reports   
Wednesday, 01 May 2013 19:31
Below is a look at some of the headlines for companies that made news in the healthcare sector on May 1, 2013.

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 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.

Also Wednesday:

("3SBio" or "the Company"), a leading China-based biotechnology company focused on researching, developing, manufacturing and marketing biopharmaceutical products, today announced that it has filed the Company's Annual Report on Form 20-F for the fiscal year ended December 31, 2012 with the U.S. Security and Exchange Commission on April 30, 2013.

Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ)
(the "Company") will announce its first quarter 2013 financial and operating results after market close on Tuesday, May 7, 2013. The Company will host a conference call to discuss these results on Wednesday, May 8, 2013, at 8:30 a.m., Eastern Time.

Allergan, Inc. (NYSE: AGN) today announced operating results for the quarter ended March 31, 2013.

Amarantus BioScience Holdings, Inc. (OTCQB: AMBS), a biotechnology company discovering and developing treatments and diagnostics for diseases associated with neurodegeneration and apoptosis, has posted to its Web site a White Paper titled “A Review of MANF Literature: Implications for the Development Program of Amarantus Bioscience Holdings.”

ArQule, Inc. (NASDAQ: ARQL)
today announced that it will report its financial results for the first quarter of 2013 on Wednesday, May 8, 2013 at 7:00 a.m. Eastern Time.

EnteroMedics Inc. (NASDAQ: ETRM)
, the developer of medical devices using neuroblocking technology to treat obesity, metabolic diseases and other gastrointestinal disorders, today announced financial results for the three months ended March 31, 2013.

Exact Sciences Corp. (Nasdaq: EXAS) today announced its financial results for the quarter ended March 31, 2013.

Genomic Health, Inc. (Nasdaq: GHDX) today announced that it will host an investor webcast and conference call to discuss the positive development and clinical validation study results of its biopsy-based Oncotype DX prostate cancer test on Wednesday, May 8, 2013 at 1:00 p.m. Pacific Time.

Guided Therapeutics, Inc. (OTCBB & OTCQB: GTHP) today announced that multi-center, clinical trial results for the LuViva® Advanced Cervical Scan are set to be published in an upcoming issue of Gynecologic Oncology, the official publication of the Society of Gynecologic Oncology.

Medgenics, Inc. (NYSE MKT: MDGN and AIM: MEDU, MEDG) (the “Company” or “Medgenics”),, the developer of a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, today announces that all resolutions proposed at the Company’s Annual Meeting of Stockholders held yesterday were duly passed.

Merck (NYSE: MRK)
, known as MSD outside the United States and Canada, today announced financial results for the first quarter of 2013.

MiMedx Group, Inc. (NasdaqCM: MDXG)
, an integrated developer, manufacturer and marketer of patent protected regenerative biomaterials and bioimplants processed from human amniotic membrane, announced today its results for the quarter ended March 31, 2013.

Novadaq® Technologies Inc. (TSX:NDQ)(NASDAQ:NVDQ)
, a developer of clinically-relevant fluorescence imaging solutions for use in surgical and outpatient wound care procedures, announced today the sale of US$50.3 million of common shares, at a price to the public of $12.90 per common share, pursuant to an underwriting agreement with Canaccord Genuity Inc.

NPS Pharmaceuticals, Inc. (Nasdaq: NPSP)
announced today that it will report its first quarter 2013 financial results on Thursday, May 9, 2013 at approximately 8:00 a.m. ET.

Oramed Pharmaceuticals Inc. (NASDAQCM: ORMP)
, a developer of oral delivery systems of drugs currently administered by injection, announced today that its article titled, "Glucose-reducing effect of the ORMD-0801 oral insulin preparation in patients with uncontrolled Type 1 diabetes: a pilot study," has been published in PLOS ONE, an international, peer-reviewed, open-access journal which covers primary scientific and medical research.

pSivida Corp. (NASDAQ:PSDV)
, a leader in developing sustained release, drug delivery products for treatment of back-of-the-eye diseases, announced today that the U.S. Food and Drug Administration (FDA) acknowledged receipt of the resubmission of the New Drug Application (NDA) for ILUVIEN® for treatment of chronic diabetic macular edema (DME) and stated that the resubmission was considered a complete, class 2 response to the FDA’s November 2011 complete response letter.

Rite Aid (NYSE: RAD)
is proud to announce its second straight year of support for one of the country's premier women's cancer run/walks, marking platinum sponsorship for both the New York and the Los Angeles events this May.

Sangamo BioSciences, Inc. (Nasdaq: SGMO)
today announced that the company will release its first quarter 2013 financial results on Wednesday, May 8, 2013, after the market closes.

, the global specialty biopharmaceutical company, today announced that the US Food and Drug Administration (FDA) approved the prescription medication Vyvanse® (lisdexamfetamine dimesylate) Capsules, (CII) as a maintenance treatment in children and adolescents with Attention-Deficit/Hyperactivity Disorder (ADHD).

Soligenix, Inc. (OTCQB: SNGX), a clinical stage biopharmaceutical company focused on developing products to treat inflammatory diseases and biodefense countermeasures where there remains an unmet medical need, announced today it will jointly develop a treatment for Melioidosis through a worldwide exclusive collaboration with Intrexon, a synthetic biology company that utilizes its proprietary technologies to provide control over cellular function.

Stevia Corp.(OTCQB: STEV) ("Stevia Corp" or the "Company"), a farm management company focused on the economic development of stevia, the fastest growing product in the alternative sweetener sector, is pleased to announce the appointment of Mr. Andrew Kaelin to its growing advisory board as a specialist in aquaculture matters.

ViroPharma Incorporated (Nasdaq: VPHM)
today announced financial results for the first quarter of 2013. Net sales were $107 million for the first quarter ended March 31, 2013 as compared to $136 million in the comparative period of 2012.

Vycor Medical, Inc. ("Vycor") (OTCBB: VYCO) announced today that it has finalized the design prototypes of two new product variations of its existing VBAS product suite to be developed specifically for endoscopic work within the ventricles of the brain.

Vycor Medical, Inc. ("Vycor") (OTCBB: VYCO)
announced today it has finalized the design of two differently-sized prototypes of its existing ViewSite™ Brain Access System ("VBAS") product suite which are specifically targeted at the pediatric neurosurgical market. It is anticipated that the new devices will be commercially available by the end of 2013 and will fall under the Company's existing U.S. FDA 510(k) clearance for VBAS.

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