World's Most Expensive Medicine

Novartis Gene Therapy to Cure Rare Disease in Infants


Source:  Novartis Basel, Switzerland

$2.125 Million Dollar Cure
The US Food and Drug Administration just approved the first gene therapy for a specific and deadly type of spinal muscular atrophy.  This is a very rare inherited disease that is detected in infants.  Novartis has priced the drug at $2.125 million or $425,000 for a five year regimen.  This raises many serious ethical questions about the cost of life saving drugs, who receives them and how to pay.

Novartis Acquisition
Novartis acquired the blockbuster drug through a $8.7 billion acquisition. They are now launching it.  It's raising a lot of issues, concerns and hope.  If Big Pharma can address this rare disease and save infant lives, can it save other lives at birth from other diseases and at what cost and profit for them?

SMA
Spinal Muscular Atrophy (SMA) is an inherited neuromuscular disease that causes progressive muscular atrophy.  The gene therapy that reverses it is Zolgensma, that uses a re-engineered virus to deliver an effective replacement for the defective gene that causes the disease.  The most severe forms of disease cause infants to die or be on breathing support by the age of two.

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