The Food and Drug Adminstration demonstrates quirky timing on occassions, and it recently announced one of its most significant drug approvals for the year just a day out from Christmas Eve.
Spinraza is the name of the drug, and it is used to treat a rare condition known as spinal muscular atrophy, which is the number one genetic cause of deaths in children and babies. The disease affects about one in 10,000 newborn babies, and manifests in a number of agonising symptions including paralysis and general debility.
The FDA’s Spinraza approval was covered extensively by the press, including The Wall Street Journal, which noted that the share price of Spinraza’s original developer, Ionis Pharmaceuticals, rose after the announcement. The drug is licensed to Biogen in Massachusetts.
Research appears to support the claim that Spinraza has demonstrable benefits. In a trial where one group of infants received a placebo and another group received the new drug, 40 per cent of the Spinraza dosed children managed to reach milestones of development such as crawling, sitting and walking. Of the children who did not receive the drug, none managed to reach these milestones. The FDA helped design the research.
Patient groups welcomed the development, saying Spinraza’s approval was to be celebrated as a force for positive change and general improvement in the lives of those suffering from spinal muscular atrophy. However the medication could cost up to $250,000 annually for each patient, which might be an issue for some insurers. The price of Spinraza is on a par with the cost of a number of drugs that are used to combat rare conditions.
Speeding up Approval
The path to taking a new drug to market can require a great deal of energy and persistence. If concerned about how long does it take to get a FDA 510k approved, it would be wise to consult experts in the field such as http://www.fdathirdpartyreview.com/.
While announcing Spinraza’s approval, the FDA also made a point of discussing its determination to speed up the process of getting drugs to patients who needed them urgently and then onto the general market. It’s thought Republicans in Congress support this move and it is likely to carry even greater importance when Donald Trump takes up his role in the Oval Office.