Miglustat (Zavesca) Receives Negative Opinion from the European Committee for Medicinal Products for Human Use for Extension of Indication for Niemann Pick Type C
ALLSCHWIL, Switzerland, October 19, 2007 - ActelionÂ announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a negative opinion for the extension of indication on the use of miglustat (Zavesca) in patients suffering from Niemann Pick type C (NP-C), a rare genetic disease associated with multiple neurological manifestations.Â
The company intends to request a re-examination of the CHMP opinion. Accordingly, the CHMP would issue a final opinion in the first quarter of 2008
About Niemann Pick type C NP-C is a rare, fatal, degenerative, genetic condition primarily affecting children and teenagers, but which can strike at any age. The symptoms are caused by the storage of some glycosphingolipids within certain cells in the body, including the brain. It is relentlessly progressive and most patients die within five to ten years of diagnosis. Neurological deterioration is a key feature of the disease, and can manifest itself as clumsy body movements, balance problems, slow and slurred speech, difficulty in swallowing, problems with eye movements and seizures. Intellectual decline is common. In the final stages of the disease the child or young adult is frequently bedridden, has little muscle control and is intellectually impaired. There is currently no treatment option for this condition.
About miglustat in Niemann Pick Type C
The regulatory review in the European Union is primarily based on the 12 and 24-month results from a randomized controlled study evaluating safety and efficacy of miglustat in 29 adult/juvenile patients suffering from Niemann Pick type C (NP-C). A non-controlled sub-study also included 12 pediatric patients for 24 months. At 12 months, although not statistically significant, the study showed trends toward improvement or stabilization in terms of saccadic eye movements (by electrophysiological assessment) and swallowing and audition (by clinical assessment) in patients receiving miglustat compared to standard of care.
About ZavescaÂ® (100 mg miglustat) and type 1 Gaucher disease ZavescaÂ® (100 mg miglustat) is indicated for the oral treatment of mild to moderate type 1 Gaucher Disease. ZavescaÂ® may only be used in the treatment of type 1 Gaucher patients for whom enzyme replacement therapy is unsuitable. It is approved in the European Union, United States, Canada, Switzerland, Brazil, Australia and Israel.
ZavescaÂ® safety information Gastrointestinal events, mainly diarrhea, have been observed in more than 80% of patients treated with ZavescaÂ®, either at the onset of treatment or intermittently during treatment. The majority of cases is mild and is expected to resolve spontaneously on therapy. In clinical practice, diarrhea has been observed to respond to diet modification (reduction of lactose and other carbohydrate intake), to taking ZavescaÂ® away from meals, and/or to anti-diarrheal medication such as loperamide. In some patients, temporary dose reduction may be necessary. Patients with chronic diarrhea or other persistent gastrointestinal events that do not respond to these interventions should be investigated according to clinical practice. ZavescaÂ® has not been evaluated in patients with a history of significant gastrointestinal disease, including inflammatory bowel disease and CF.
Peripheral neuropathy has been reported in type 1 Gaucher patients treated with ZavescaÂ®. Patients should undergo a neurological exam at the start of treatment and regularly thereafter. ZavescaÂ® should be reassessed in patients who develop symptoms of peripheral neuropathy. ZavescaÂ® may cause fetal harm if administered to a pregnant woman and is contraindicated in women who are or who may become pregnant; patients should be apprised of the potential hazard to the fetus. There is a risk of impaired fertility in men. Men should maintain reliable contraceptive methods and not plan to conceive while taking ZavescaÂ® and for three months thereafter.
Actelion Ltd Actelion Ltd is a biopharmaceutical company with its corporate headquarters in Allschwil/Basel, Switzerland. Actelion's first drug TracleerÂ®, an orally available dual endothelin receptor antagonist, has been approved as a therapy for pulmonary arterial hypertension. Actelion markets TracleerÂ® through its own subsidiaries in key markets worldwide, including the United States (based in South San Francisco), the European Union, Japan, Canada, Australia and Switzerland. Actelion, founded in late 1997, is a leading player in innovative science related to the endothelium - the single layer of cells separating every blood vessel from the blood stream. Actelion focuses on the discovery, development and marketing of innovative drugs for significant unmet medical needs. Actelion shares are traded on the SWX Swiss Exchange (ticker symbol: ATLN).