ZOLGENSEMA - What makes this drug so valuable?

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Spinal Muscular Atrophy (SMA)

What is spinal muscular atrophy?

Source: medicalnewstoday

SMA is a genetic disorder that affects the central nervous system, peripheral nervous system, and voluntary muscle movement (skeletal muscle).  The majority of nerve cells that govern muscles are found in the spinal cord, which explains why the disease's name includes the word spinal. The primary effect of SMA is on muscles, which do not receive impulses from these nerve cells. When muscles aren't stimulated by nerve cells, they atrophy, which is a medical term for shrinking.

SMA is a motor neuron disease that causes the loss of nerve cells called motor neurons in the spinal cord. The most common form of SMA (chromosome 5 SMA, also known as SMN-related SMA) has a wide range of onset, symptoms, and progression rates. Types 1 through 4 of chromosome 5-related SMA, which is commonly autosomal recessive, are categorised to account for these variances.

The age at which SMA symptoms appear generally coincides with the severity of motor function impairment: The stronger the influence on motor function, the younger the onset age. Children who show symptoms at birth or early in childhood usually have the lowest degree of functioning (type 1). SMA that develops later in life and has a milder course (types 2 and 3, and type 4 in teenagers or adults) is associated with higher levels of motor function.

Zolgensma (onasemnogene abeparvovac-xioi), the first gene-replacement therapy for a neuromuscular condition, was authorised by the FDA in May 2019. Zolgensma is a one-time intravenous (into the vein) infusion for the treatment of juvenile patients with SMA who have bi-allelic mutations in the SMN1 gene and are presymptomatic at the time of diagnosis.

What exactly is ZOLGENSMA?

ZOLGENSMA is a prescription gene therapy used to treat spinal muscular atrophy in children under the age of two (SMA). ZOLGENSMA is administered as a one-time intravenous infusion. ZOLGENSMA’s characteristics include:

  • This is a one-time-only dose.
  • Intravenously administered (IV)
  • This is a 60-minute infusion.

By replacing the function of the missing or nonworking survival motor neuron 1 (SMN1) gene, ZOLGENSMA is designed to tackle the genetic root cause of SMA with a one-time-only dose. Motor neuron cells are instructed to create more survival motor neuron (SMN) protein by the new gene. SMN protein is required for motor neuron cell survival and muscle function. Motor neuron cells become weaker with each passing day without treatment, eventually losing all function and dying. Breathing, eating, swallowing, and speaking become difficult to impossible as a result. The illness is likely to become life-threatening if these cells quit operating.

ZOLGENSMA is designed to deliver a new, working copy of the SMN gene to produce SMN protein, which preserves motor neuron cells throughout the body. In turn, ZOLGENSMA stops the progression of SMA and sustains the remaining muscle function needed for children to survive. The earlier treatment with ZOLGENSMA is given, the sooner muscle function may be preserved. ZOLGENSMA is not a cure and cannot reverse the damage already caused by SMA before treatment.

What is the most crucial information regarding ZOLGENSMA that I should be aware of?

  • ZOLGENSMA might result in severe liver damage. In children who get ZOLGENSMA, liver enzymes may become high, indicating immediate severe liver injury.
  • Before and after receiving ZOLGENSMA, patients will be given an oral corticosteroid and will have regular blood tests to check their liver function.
  • If the patient's skin and/or whites of the eyes become yellowish, or if the patient misses or vomits a corticosteroid dose, contact the patient's doctor right away.

What do I need to know about ZOLGENSMA vaccinations?

  • Consult the patient's doctor to see whether any changes to the immunisation schedule are required to accommodate the corticosteroid medication.
  • RSV (respiratory syncytial virus) protection is advised.

Is it necessary for me to take special measures with the patient's body waste?

Small levels of ZOLGENSMA may be discovered in the patient's faeces for a short time. For the first month after receiving ZOLGENSMA, keep your hands clean when in close touch with bodily waste. Disposable diapers should be sealed in trash bags and discarded with ordinary garbage.

What are the side effects of ZOLGENSMA that might occur?

Elevated liver enzymes and vomiting were the most common negative effects experienced by persons taking ZOLGENSMA.

So, what makes ZOLGENSMA so expensive?

Source: funtrafoo

For children under the age of two years with spinal muscular atrophy (SMA), Zolgensma is a one-time-only gene therapy procedure that costs $2.1 million. Because it "dramatically alters the lives of families impacted by this horrible disease" and the claimed expense of bringing new treatments to market, Novartis has judged that Zolgensma is worth that much money. However, there is some debate over this price.

For starters, the National Institutes of Health and several charities are dedicated to finding treatments for SMA, including Sophia's Cure, Cure SMA, Getty Owl Foundation, Fighting SMA, Jadon's Hope Foundation, the Gwendolyn Strong Foundation, and Miracle for Madison, funded the early development of Zolgensma. Many of these organisations rely on donations from patients' relatives and friends to fund research and clinical trials for novel SMA treatments.

Vas Narasimhan, the CEO of Novartis, claims that gene treatments are a medical breakthrough because they provide the possibility of a single-dose solution for lethal hereditary disorders. In some circumstances, a multi-dose treatment with incremental improvements is a viable option. Spinraza, for example, is a Zolgensma alternative that is taken four times a year for the rest of one's life. The list price is $750,000 for the first year and then $350,000 per year after that, for a total of almost $4 million over ten years.

But how do they come up with the final price? Many firms adopt a value-based pricing approach, which determines the cost based on the number of years of life gained as well as the drug's efficacy. However, this entails determining the value of life and ensuring that the firm gets a sufficient profit on their investment. The full cost of bringing a new medication to market is rarely disclosed, and Novartis didn't develop Zolgensma; instead, it bought it for $8.7 billion from AveXis in the United States.

Most health insurance plans do not cover such expensive one-time procedures, and a high price tag is no guarantee that a drug will meet all of your expectations. Novartis has agreed to accept payments over five years at a rate of $425,000 per year and has stated that it will offer partial refunds if the treatment fails.


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