Health Care

FDA Approves First Gene Therapy for Spinal Muscular Atrophy

FDA Approves First Gene Therapy for Spinal Muscular Atrophy”

Called Zolgensma, it is a type of cutting-edge, one-time treatment called a "gene therapy" that treats the disease spinal muscular atrophy at its genetic root. The dose administered is decided on based on the weight of the patient.

Around 10,000 to 25,000 USA children and adults are estimated to have the disease, which occurs in one of every 6,000 to 10,000 births.

According to David Lennon of AveXis, there are now around 1,100 patients eligible for treatment with Zolgensma in the US. One version the Energy and Commerce Committee is considering would exclude rare disease treatments from those requirements.

Given the severity of the condition, and Zolgensma's early results, Pearson said insurers would going "cover Zolgensma no matter the price" even when Novartis has publicly floated the idea of a price approaching $5m. Most do not survive past early childhood. The gene encodes the survival motor neuron (SMN) protein - a protein found throughout the body, which is critical for the maintenance and function of specialised nerve cells, called motor neurons.

Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, stated that patients with SMA "now have another treatment option to minimise the progression of SMA and improve survival". Patients treated with the drug also showed significant improvement in their ability to reach developmental motor milestones, such as head control and sitting without support, compared to the natural history of patients with the disease. The company noted that it would provide rebates to insurance companies if the drug is not successful, though it did not offer details about what would be considered failure.

CEO Vas Narasimhan recently signaled that the therapy could be worth more than $5 million, considering the years of intensive care the drug can replace. "It is a positive outcome for patients and the entire health system that Novartis instead chose to price Zolgensma at a level that more fairly aligns with the benefits for these children and their families".

The Institute for Clinical and Economic Review, an independent watchdog group, said that the price of Zolgensma "falls within the upper bound" of its value-based benchmark range, which takes into account the value of increased lifespans.

"The acquisition price was based not on cost to develop the drug but based on expectations of high returns", he said.

This treatment is a breakthrough for Spinal Muscular Atrophy (SMA) patient and expected to be a great relief for patients however; though Novartis claimed it to be cost effective as compared to 10-year cost of current chronic SMA treatment (estimated at United States dollars 4.1 million).But this unique treatment is priced at a whooping price tag of USD 2.125 Million.

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