Meet the World’s most expensive drug: Zolgensma

The U.S. FDA recently approved the much talked about Novartis’s drug Zolgensma for treating a life-threatening condition Spinal Muscular Atrophy (SMA).  SMA is a neuromuscular disorder that worsens the muscles over time. It happens due to a mutation in the SMN1 gene that results in the deficiency of an SMN protein, which is essential for the functioning and survival of motor neurons. SMA is a leading genetic cause of death in infants. With time the disease leads to difficulties in breathing, paralysis and even death of the babies born with Type 1- the most severe type of SMA.
Zolgensma is a gene therapy that is designed to deliver a working SMN1 gene to facilitate the transcription of functional SMN protein for better functioning of motor neurons. The drug is enveloped by a shell of a genetically engineered virus, the adeno-associated virus (AAV) 9. The SMN1 gene in Zolgensma comprises a double-stranded DNA, which is responsible for producing a standard copy of SMN1 to motor neurons, which further produces healthy SMN protein. It also has a transgene that ensures the production of SMN protein is sustainable and life-long.

Zolgensma, priced at USD 2.125 million one time, and USD 425,000 per year for five years, is the costliest drug present in the market. Approved for the treatment of children under the age of 2 years, including those who have not yet started to show symptoms, Novartis claims to have treated around 150 patients with Zolgensma. Without a doubt, Novartis is going to face many backlashes from the critics for pricing a medicine several thousands of dollars. The availability of the life-saving drug is useless if it is not affordable.  However, Novartis defends itself, claiming Zolgensma is a one-time treatment and is more effective than other available ones that are long-term therapies. 
The cost factor is not the only hurdle Zolgensma has to cross. There is also a competition. Zolgensma is going to compete with Biogen’s Spinraza which got approved in 2016 for the treatment of all types of SMA. Zolgensma may have the upper hand as it is a one-time treatment unlike Spinraza, which requires regular spinal infusion worth USD 750,000 on the first year and USD 375,000 annually life-long. Roche is not far behind. The drugmaker is also developing a pill for SMA known as risdiplam to set its foot in the Spinal Muscular Atrophy market by 2020.

Although, clinicians trust the lifelong treatments as promised by Zolgensma. However, it is still too early to say that. Many researchers are also stressing upon the need for early Spinal Muscular Atrophy diagnosis. Early diagnosis is crucial to provide infants with the right treatment at the right time. Thus, Novartis is focusing on genetic testing of the newborn at the time of their birth.