DUBLIN, IRELAND--(Marketwired - Oct 3, 2014) - Horizon Pharma plc (NASDAQ: HZNP), a specialty biopharmaceutical company with a portfolio of products in arthritis, inflammation and orphan diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for ACTIMMUNE® (Interferon gamma-1b) for the treatment of Friedreich's ataxia (FA), a rare, genetic disease for which there are currently no approved treatments.
The U.S. Orphan Drug Act (ODA) provides for granting special status to a drug or biological product to treat a rare disease or condition that affects fewer than 200,000 patients in the United States. Orphan designation qualifies the sponsor of the drug for various development incentives, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan-drug designation is not subject to the Prescription Drug User Fee Act (PDUFA) fees. The designation may provide ACTIMMUNE, if approved for the treatment of FA, with seven years of market exclusivity for this indication.
FA is a debilitating, life-shortening, degenerative neuro-muscular disorder that affects about one in 50,000 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.
"This orphan-drug designation for ACTIMMUNE is an important regulatory advancement as we build our development program in FA, a rare, debilitating disorder with no currently approved treatments," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "We look forward to the release of results from a Phase 2 study sponsored by the Friedreich's Ataxia Research Alliance (FARA) and conducted at Children's Hospital of Philadelphia."
ACTIMMUNE is currently approved for two rare diseases in the United States. It is approved by the FDA to reduce the frequency and severity of serious infections associated with Chronic Granulomatous Disease (CGD), a genetic disorder that affects the functioning of a type of white blood cell of the immune system, neutrophils or phagocytes, leading to recurrent severe bacterial and fungal infections and chronic inflammatory conditions. In addition, ACTIMMUNE is approved by the FDA to slow the worsening of severe, malignant osteopetrosis (SMO), a genetic disorder, that affects normal bone formation causing the abnormal accumulation of bone material which tends to narrow the space inside bones where bone marrow is formed. This can cause failure of the bone marrow, leading to a decrease in various blood cells such as red blood cells and white blood cells. For more information, please visit www.ACTIMMUNE.com.
Important Safety Information
ACTIMMUNE is contraindicated in patients who develop or have known hypersensitivity to interferon-gamma, E. coli-derived products, or any component of the product.
The most common adverse experiences occurring with ACTIMMUNE therapy are "flu like", or constitutional symptoms such as fever, headache, chills, myalgia, or fatigue, which may decrease in severity as treatment continues. Some of the "flu-like" symptoms may be minimized by bedtime administration of ACTIMMUNE. Acetaminophen may be used to prevent or partially alleviate the fever and headache.
Reversible neutropenia and thrombocytopenia have been observed during ACTIMMUNE therapy. Caution should be exercised when administering ACTIMMUNE in patients with myelosuppression or in combination with other potentially myelosuppressive agents. ACTIMMUNE may also depress hepatic metabolism of certain drugs that are metabolized by the hepatic cytochrome P-450 system. ACTIMMUNE has not been tested for carcinogenic potential. ACTIMMUNE is pregnancy Category C and it is unknown if ACTIMMUNE is excreted in human milk.
Reversible elevations of AST and/or ALT have been observed during ACTIMMUNE therapy. Patients begun on ACTIMMUNE therapy before one year of age should receive monthly assessments of liver function. If severe hepatic enzyme elevations develop, ACTIMMUNE dosage should be modified.
At doses 10 times greater than the weekly recommended dose, ACTIMMUNE may exacerbate pre-existing cardiac conditions or may cause reversible neurological effects such as decreased mental status, gait disturbance and dizziness. Therefore, caution is advised when ACTIMMUNE is administered to patients with seizure disorders or compromised CNS function or when administered to patients with cardiac conditions such as ischemia, heart failure or arrhythmia.
For more information on ACTIMMUNE, please see the Full Prescribing Information at www.ACTIMMUNE.com.
About Horizon Pharma plc
Horizon Pharma plc is a specialty biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated products that address unmet medical needs. The company markets a portfolio of products in arthritis, inflammation and orphan diseases. The Company's U.S. marketed products are ACTIMMUNE® (interferon gamma-1b), DUEXIS® (ibuprofen/famotidine), RAYOS® (prednisone) delayed-release tablets and VIMOVO® (naproxen/esomeprazole). Horizon's global headquarters are in Dublin, Ireland. For more information, please visit www.horizonpharma.com.
This press release contains forward-looking statements, including statements regarding the potential implications of Orphan Drug designation for ACTIMMUNE in FA, results of a Phase 2 study with ACTIMMUNE and the Company's future development plans for ACTIMMUNE in FA. 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 Horizon will be able to realize the anticipated benefits of Orphan Drug designation, whether the results of the Phase 2 clinical trial of ACTIMMUNE in FA will warrant further development, and Horizon's ability to obtain regulatory approval for and successfully commercialize ACTIMMUNE for the treatment of FA. For a further description of these and other risks facing the Company, please see the risk factors described in the Company'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 the Company undertakes no obligation to update or revise these statements, except as may be required by law.