Zavesca/Miglustat update from US FDA Panel shows some hope for Australia

UPDATE 2-US FDA panel backs new use for Actelion drug
Tue Jan 12, 2010 5:31pm

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* FDA decision due by March 10
* Agency usually follows panel recommendations (Adds panelist, company comments)
By Lisa Richwine
SILVER SPRING, Md., Jan 12 (Reuters) - A U.S. advisory panel on Tuesday recommended approval of an Actelion Ltd (ATLN.VX) drug for treating a rare disease that often kills patients in their teens after fats accumulate in their organs.
A Food and Drug Administration advisory panel voted 10-3 to recommend approval of Actelion's Zavesca pill for patients with a condition called Niemann-Pick type C disease (NP-C).
Europe's largest biotech company already sells Zavesca in the United States for another rare disorder, type 1 Gaucher disease.
Panel members who supported the drug said there were signs Zavesca would help patients with NP-C even though the drug failed to meet the main effectiveness goal in a company study.
"The risk from the drug is not devastating, but the disease is devastating. I think patients deserve an opportunity to see if they can benefit," Katherine Flegal, a senior research scientist at the Centers for Disease Control and Prevention, said.
Actelion is trying to cut its dependence on Tracleer, a drug that treats a rare heart and lung disorder and brings in more than $1 billion a year. Zavesca sales through the first nine months of 2009 were 38.3 million Swiss francs ($37 million).
Doctors can prescribe Zavesca now for NP-C, but Actelion needs FDA clearance to market the drug specifically for that use. Patient advocates also said insurers are reluctant to pay for the drug for NP-C patients without the approval. The drug costs $159,000 a year per patient.
NP-C, which has no approved treatment in the United States, causes cholesterol and other fats to collect in the brain and various organs. That leads to impaired brain function, lack of muscle control, slurred speech and eventually death. Symptoms usually develop around age 10, and most patients die within five to 10 years of diagnosis.
Just 500 patients worldwide are known to have the disease, including about 200 in the United States, Actelion said.
Zavesca, or miglustat, was designed to block the action of an enzyme involved in producing the fats that cause problems in NP-C.
But the drug failed to meet the main goal in a 29-patient study comparing it to a placebo on measurements of eye movements that Actelion said were a sign of the disease's progression.
The company argued that other findings from surveys of NP-C patients who had been treated with Zavesca showed the drug slowed or stabilized problems with swallowing, walking and speaking.
That type of data is considered less reliable than findings from clinical trials. Actelion, however, said the improvements showed the drug was working because NP-C patients without treatment will get worse over time.
"This is a relentlessly progressive disease," Dr. Isaac Kobrin, Actelion's chief medical officer, told the panel.
"We clearly see a change in the progression rate (with Zavesca). It's a very clear signal," he said.
Actelion said risks such as diarrhea were manageable and outweighed by benefits. FDA reviewers said some NP-C patients had reduced growth and lower levels of blood platelets. Actelion said it would urge monitoring of those issues.
The FDA usually follows panel recommendations when deciding whether to approve medicines. A decision is due by March 10.