By: Kevin Brady, Esq. In May of this year, the Federal Drug Administration (FDA) approved the most expensive drug in the world, Zolgensma. The drug was developed by Swiss drug maker, Novartis, and costs $2.1 million for the one-time single dose. The drug maker will spread the burden of the high cost by allocating payments for the drug over a 5- year plan at $425,000 a year. Zolgensma is a new gene therapy drug used to treat spinal muscular atrophy (SMA). SMA is a rare, genetic neuromuscular disease caused by a defective or missing gene. Infants with the missing or defective gene will lose motor function control and are likely to lose the ability to breath, speak, swallow and walk. Essentially, Zolgensma can be used to treat all types of SMA in newborns and toddlers up to age two (2). Not only is this the most expensive drug in the world, the drug maker, Novartis, has recently come under scrutiny by the FDA for allegedly manipulating pre-clinical data prior to FDA approval. For now, the FDA is not inclined to take Zolgensma off the market, as they still believe in the safety and efficacy of the drug. However, the FDA is likely to take action against the drug maker, most likely in the form of civil and criminal penalties. With all the scrutiny around this new drug, plan sponsors should be aware of the high-price tag associated with this drug and the alleged misrepresentations of data by the drug maker in its seeking of FDA approval. Plan sponsors should carefully consider their options when drafting their plans. Gene Therapy is always a hot topic as it is typically associated with a high cost. For Zolgensma, while there may be a high up-front cost, if the drug is effective, most importantly, it will save lives and potentially years of expensive, demanding, and less effective alternative treatments.