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DUBLIN, IRELAND -- (Marketwired) -- 06/05/15 -- Horizon Pharma plc (NASDAQ: HZNP), a biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, today announced it has initiated the Phase 3 S
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"As a rare disease with no currently approved treatments, Friedreich's Ataxia represents an area of significant unmet medical need," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "The Phase 3 study represents a significant next step in evaluating ACTIMMUNE in this population, which is approximately four thousand people in the United States. We look forward to continuing to work with the FDA with the goal of providing a potential treatment option to the FA community."
This Phase 3 trial (NCT02415127) is a randomized, multi-center, double-blind, placebo-controlled study with patients randomized 1:1 to receive subcutaneous doses of either ACTIMMUNE or placebo three times a week for a total of 26 weeks. Approximately 90 patients will be enrolled at four sites in the United States. The primary endpoint will measure the change in neurological outcome and evaluate the effect of ACTIMMUNE versus placebo as measured by the modified Friedreich's Ataxia Rating Scale (mFARS), focused on objective neurologic measures such as upper and lower extremity coordination improvement from baseline. FARS is used to measure neurological signs associated with FA, with higher scores reflecting a greater level of disability.
In addition to safety and efficacy, the STEADFAST trial will evaluate the pharmacokinetic characteristics of ACTIMMUNE in people with FA.
"This clinical trial is an example of the progress the scientific community has made in understanding FA," said Ronald J. Bartek, president, director and co-founder, Friedreich's Ataxia Research Alliance (FARA). "Less than 20 years ago, we had not identified the cause of FA, and today we have a late-stage clinical study that brings us one step closer to potentially having the first treatment for people with FA."
In April 2015, ACTIMMUNE was granted Fast Track status for FA by the U.S. Food and Drug Administration (FDA). This designation provides greater access to and more frequent communication with the FDA throughout the entire drug development and review process, with the goal of possibly expediting approval. Fast Track designation also gives Horizon Pharma the opportunity to potentially submit sections of the ACTIMMUNE registration dossier for FA on a rolling basis, and allows ACTIMMUNE to be considered for priority review at the time of submission based on forthcoming clinical data.
About Friedreich's ataxia (FA)
FA is a debilitating, life-shortening and degenerative neuro-muscular disorder that affects approximately 4,000 people in the United States. Onset of symptoms can vary from five years old to adulthood, with the childhood onset tending to be associated with a more rapid progression. A progressive loss of coordination and muscle strength leads to motor incapacitation and often the full-time use of a wheelchair. Most young people diagnosed with FA require mobility aids such as a cane, walker or wheelchair by their teens or early 20's. There are currently no approved treatments for FA.
The Friedreich's Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), non-profit, tax exempt organization dedicated to curing Friedreich's ataxia (FA), a rare neuromuscular disorder, through research. For more information about FA, visit the Friedreich's Ataxia Research Alliance (FARA) website at www.curefa.org.
ACTIMMUNE (interferon gamma-1b) is a biologically manufactured protein similar to one the body makes naturally to help prevent infection. ACTIMMUNE is currently approved by the U.S. Food and Drug Administration (FDA) for use in two rare diseases. It is indicated to reduce the frequency and severity of serious infections associated with Chronic Granulomatous Disease (CGD), a genetic disorder that affects the functioning of some cells of the immune system. In addition, ACTIMMUNE is indicated to slow the worsening of severe, malignant osteopetrosis (SMO), a genetic disorder that affects normal bone formation. For more information, please see www.ACTIMMUNE.com.
Indications and Usage
Chronic Granulomatous Disease (CGD)
ACTIMMUNE is approved by the U.S. FDA to reduce the frequency and severity of serious infections associated with CGD. CGD is a genetic disorder that affects the functioning of some cells of the immune system.
Severe, Malignant Osteopetrosis (SMO)
ACTIMMUNE is approved by the U.S. FDA to slow the worsening of SMO. SMO is also a genetic disorder that affects normal bone formation.
Important Safety Information
The most common side effects with ACTIMMUNE are "flu like" symptoms, such as fever, headache, chills, myalgia (muscle pain), or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may minimize these symptoms. Acetaminophen may be helpful in preventing fever and headache.
ACTIMMUNE can cause severe allergic reactions and/or rash. Do not use ACTIMMUNE if you are allergic to interferon-gamma, E. col-derived products, or any component of the product. (See Full Prescribing Information for a list of components). If you develop a serious reaction to ACTIMMUNE discontinue it immediately and contact your doctor or seek medical help.
At high doses, ACTIMMUNE can cause "flu like" symptoms, which may worsen some pre-existing heart conditions. Tell your doctor if you have a cardiac condition, such as irregular heartbeat, heart failure, or decreased blood flow to your heart.
ACTIMMUNE may cause reversible changes to your nervous system, including decreased mental status, walking disturbances, and dizziness. Tell your doctor if you have a history of seizures or other neurological disorders.
Bone marrow function may be suppressed with ACTIMMUNE and decreased production of cells important to the body may occur. This effect, which can be severe, is usually reversible when the drug is discontinued or the dose is reduced. Tell your doctor if you have, or have had reduced bone marrow function. Your doctor will monitor these cells with blood tests at the beginning of therapy and at 3 month intervals thereafter.
Taking ACTIMMUNE may cause reversible changes to your liver function, particularly in patients less than one year old. Your doctor will monitor your liver function at the beginning of therapy and at 3 month intervals thereafter.
If you are pregnant or plan to become pregnant or plan to nurse you should consult your physician.
If you are receiving ACTIMMUNE at home, your doctor will provide to you or your caregiver appropriate instructions on the administration of the drug and disposal of the container, needles and syringes.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
This information is not intended to replace discussions with your doctor. For additional information about ACTIMMUNE, please consult the Full Prescribing Information and the Information for the Patient/Caregiver and talk to your doctor. ACTIMMUNE is available by prescription only.
Visit www.ACTIMMUNE.com to download a copy of the ACTIMMUNE Full Prescribing Information.
About Horizon Pharma plc
Horizon Pharma plc is a biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs. The Company markets seven medicines through its orphan, primary care and specialty business units. Horizon's global headquarters are in Dublin, Ireland. For more information, please visit www.horizonpharma.com. Follow @HZNPplc on Twitter or view careers on our LinkedIn page.
Forward Looking Statements
This press release contains forward-looking statements, including statements regarding expected parameters of the Phase 3 trial of ACTIMMUNE in Freidreich's Ataxia, the potential benefits of Fast Track designation, and the potential for ACTIMMUNE as a treatment for Friedreich's ataxia. Forward-looking statements speak only as of the date of this press release and Horizon does not undertake any obligation to update or revise these statements, except as may be required by law. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release and actual results may differ materially from those in these forward-looking statements as a result of various factors. These factors include, but are not limited to, risks regarding whether results of subsequent studies will be consistent with results of prior studies, whether Horizon will conduct further studies of ACTIMMUNE or have the financial resources to do so, whether Horizon realizes any of the potential benefits of Fast Track designation, and the risks associated with pre-clinical and clinical development of drug candidates. For a further description of these and other risks facing Horizon, please see the risk factors described in Horizon's filings with the United States Securities and Exchange Commission, including those factors discussed under the caption "Risk Factors" in those filings. Forward-looking statements speak only as of the date of this press release and Horizon undertakes no obligation to update or revise these statements, except as may be required by law.
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