More drugs being approved for rare diseases in kids

NEW YORK (Reuters Health) - A growing number of drugs are coming to market to help treat rare diseases in children, a new U.S. government study finds.

Officials say the findings, reported in the journal Pediatrics, suggest that a U.S. law known as the Orphan Drug Act is working as intended.

"Orphan" drugs are called that because they target uncommon diseases that have traditionally been neglected by drug companies -- since developing treatments for rare conditions is a generally unprofitable endeavor.

The Orphan Drug Act was passed in 1983 to offer companies tax credits, marketing rights and other incentives to focus on rare diseases.

In the new study, officials at the U.S. Food and Drug Administration (FDA) found that between 2000 and 2009, there were more than three dozen orphan drug approvals for rare disorders affecting children and teenagers.

And pediatric diseases are accounting for a growing proportion of those approvals.

In the first half of the 2000-2009 decade, just 10 of 57 orphan drug approvals were for pediatric conditions. In the latter half, that rose to 28 of 91, the FDA found.

In all, there were 38 approvals for rare pediatric conditions -- though that sometimes meant a single drug got several approvals for different uses.

Disorders affecting the hormonal and metabolic systems accounted for the biggest share of approvals (39 percent).

For example, somatropin -- a synthetic form of human growth hormone -- won several different approvals over the decade. The drug got the OK to treat short stature and growth failure due to small birth size and certain rare genetic conditions, like Prader-Willi syndrome and Noonan syndrome.

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More drugs being approved for rare diseases in kids