A Fatal Muscular Disease Curable Now After FDA Approves Spinraza – A Highly Costly Drug

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The drug to treat patients with spinal muscular atrophy has won the approval of FDA. This is a giant leap of medical towards an efficient treatment of fatal diseases. This disease in its severe forms claims the life of infants before they are two years old.

Dr. Mary K. Schroth, a lung specialist in Madison Wis. has called the drug a "miracle" and a "life-changing event" while commenting on FDA approval of the drug. The new drug will change the course of the disease.

10,000 kids every year are born with spinal muscular atrophy and 400 of these are in USA. It is one of the leading genetic causes of mortality in babies. The disease creates a genetic flaw that obstacles protein production and motor neurons are not supported.

The ultimate result is muscle atrophy. Spinraza treats the underlying cause of the sickness and enables a backup gene to produce more of the necessary protein.

About 1 in 10,000 babies are born with spinal muscular atrophy or about 400 a year in the United States and it is among the leading genetic causes of death in infants. People with the disease have a genetic flaw that makes them produce too little of a protein that supports motor neurons, leading muscles to atrophy. Spinraza addresses the underlying genetic cause of the disease and enables a backup gene to produce more of the necessary protein.

The drug named "Spinraza" is not a cheap option but it is the world's most expensive drug - more than cancer treatment. Biogen is licensing the drug from Ionis Pharmaceuticals that one dose will cost $125,000 which means $625,000 to $750,000 for 5 to 6 doses in the first year. After that, three doses a year would cost $375,000 and the patients will need to take it for the rest of their lives.

Geoffrey C. Porges, an analyst for Leerink Partners comments on the high price of the drug that a storm of criticism is going to rise including presidential tweets and the insurance companies will likely limit the insurance of the drug for severely affected patients like infants only although, the drug is approved by FDA for all patients diagnosed with spinal muscular atrophy, reports New York Times.

 Ligia Del Bianco, the spokeswoman for Biogen says that the price of the drug in view of many factors like the clinical value and cost to the health care. She also added that the talks are in their initial stage with health insurance companies and the insurers' have shown a positive response to the effective drug.

Del Bianco also vowed that no patient will be deprived of treatment because of financial strain or insurance status. Biogen is working to provide financial assistance to patients and help them navigate insurance approvals.