Zolgensma: World’s Most Expensive Drug

July, 2019

Recently in 2019, the US FDA has approved Zolgensma, by Novartis, for the treatment of a neuromuscular disorder known as Spinal Muscular Atrophy (SMA). The mutation of SMN1 gene results in the deficiency of an SMN protein, which is vital for the survival and proper functioning of motor neurons. SMA is a foremost genomic reason for demise in newborns. Gradually, the infants suffering from SMA experience difficulties in breathing and can get paralyzed & lead to death.

Zolgensma is a kind of gene therapy, especially premeditated to deliver a functioning SMN1 gene to enable better functioning of motor neurons. A genetically engineered virus, also known as adeno-associated virus (AAV), encloses the drug within. The double-stranded DNA in the SMN1 gene of Zolgensma drug helps to generate a standard copy of SMN1 to produce SMN protein.

Zolgensma is a one-time treatment, which costs $2.125 million, and $425,000 every year for the next half decade. The drugs intended to treat the children below the age of 2 years can also be used for the treatment of those who have not yet shown the symptoms of the condition. Novartis claims that Zolgensma has treated approximately 150 patients suffering from SMN. Certainly, Novartis is likely to face criticism for the pricing of the drug as it is not affordable by many people.

Another hurdle, along with the high cost, for Zolgensma is the intense competition against Biogen’s Spinraza. Unlike Spinraza, which involves regular spinal infusion costing $750,000 during the first year and $375,000 yearly for the rest of the life, Zolgensma is a one-time treatment, which is an advantage for Novartis. Roche is also not very far behind and is developing a pill by name Risdiplam to enter the Spinal Muscular Atrophy market by 2020.

There has been an increased focus by researchers on the early diagnosis of Spinal Muscular Atrophy. Early diagnosis is critical to make available right treatment to infants as early as possible. Hence, Novartis is concentrating on genetic testing of the infants during the time of birth.

– Rikitha K Murthy
Healthcare – Research Analyst
Infoholic Research