Knesset committee demands inclusion of 'orphan disease' drug

"Orphan drug" is the first-ever treatment for Pompe disease, a rare but serious disease diagnosed in babies.

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July 3, 2006 22:45
1 minute read.
myozyme 88

myozyme 88. (photo credit: )

A demand to include medications for rare "orphan" disorders such as Pompe's disease - which reportedly affects seven Israeli children - in the basket of health services was sounded by the Knesset Labor, Social Affairs and Health Committee on Monday. The committee, headed by Gil Pensioners Party MK Moshe Sharon, called on Health Minister Ya'acov Ben-Yizri to speed up registration of a drug called Myozyme, which was approved by the US Food and Drug Administration in April. This "orphan drug" is the first-ever treatment for Pompe disease, a rare but serious disease diagnosed in babies (its infantile form) or older people (late-onset) that drastically reduces their muscle and respiratory function. The drug could save the children's lives, the committee said. The committee discussion was initiated by MK Muhammad Barakeh, as some of the affected children are Arab and because of consanguinity (inbreeding or marriage of cousins). An enzyme called acid alpha-glucosidase, essential for normal muscle development, is missing or deficient in victims of Pompe disease, a inherited neuromuscular condition that often kills babies. Myozyme is given by intravenous infusion into a vein. Pompe disease is one of 40 genetic diseases known as lysosomal storage disorders and was first described in 1932 by Dutch physician J.C Pompe. who observed an infant with severe muscle weakness, an oversized tongue and a very enlarged heart but normal mental development. The Knesset committee also expressed its regrets that the ministry has done nothing to advance the treatment of "orphan" diseases by giving incentives, as in the US, to drug companies that research them and manufacturer drugs to treat them. Members called on Ben-Yizri to set criteria for defining such diseases in Israel and find a way to include relevant drugs in the basket of health services supplied by the health funds at nominal cost. Etti Semama, a Health Ministry representative, said at the meeting that Myozyme was presented for approval by the ministry's pharmaceutical registration department in June. The annual cost for treating a single child is 650,000 euros.


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