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The United States Food and Drug Administration (FDA) has approved onasemnogene abeparvovec-xioi (Zolgensma, AveXis Inc, a Novartis group), the first gene therapy for children under 2 years of age with spinal muscular atrophy (SMA).
A single injection, however, will cost more than $ 2 million, making it the world's most expensive drug. According to the company, the annualized cost of Zolgensma is $ 425,000 a year for five years. The company said it is working closely with payers to create five-year results-based agreements and new payment options over time.
SMA is a rare genetic condition that can lead to paralysis, breathing difficulties and death. This is the leading genetic cause of death in infants.
"Children with ADS have difficulty fulfilling the essential functions of life.Most children with this condition do not survive because of respiratory failure," said Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research. Release.
"ADS patients now have another treatment option to minimize the progression of ADS and improve their survival.This approval reflects the current momentum of this promising new area of medicine and FDA commitment. to support and accelerate the development of these products, "said Marks.
SMA is caused by a mutation in the survival motor neuron 1 (SMN1) the gene. The gene codes for the SMN protein essential for maintaining and functioning motor neurons.
Zolgensma is a gene therapy based on an adeno-badociated virus vector that attacks the underlying cause of AMS by replacing the defective or missing virus. SMN1gene to stop the progression of the disease. A single single intravenous infusion results in the expression of the motor neurons of the SMN protein, which improves muscle movement and function as well as survival. The dosage is based on weight.
In the phase 3 STR1VE study, Zolgensma was badociated with prolonged event-free survival, motor function increase, and significant progress in patients with type 1 ADS (acute infant form).
In the START study, patients treated with Zolgensma met with motor milestones never seen in the natural history of the disease, including sitting, talking and some walking patients, with no negative effects around 4 years after the administration of the dose, according to a press release from the company. .
"In the START clinical trial we conducted with Zolgensma, all the children were alive at the end of the study and many were able to sit, roll, crawl, play and even to walk, "said Jerry Mendell, of the Gene Therapy Center in the Abigail Wexner Research Institute of the Nationwide Children's Hospital in Columbus, Ohio, said in the release.
"This level of effectiveness, administered as a single, unique treatment, is truly remarkable and offers an unprecedented level of hope to families dealing with Type 1 ADS. We now have data from of the test, 4 years later, and we are seeing the sustainability of this gene therapy, "said Mendell.
The most common side effects with Zolgensma are elevated liver enzymes and vomiting. Zolgensma contains a warning box indicating that serious severe liver injury may occur. Patients with pre-existing hepatic impairment may be at increased risk for severe liver injury. A clinical examination and laboratory tests to badess liver function should be performed prior to treatment with Zolgensma. The liver function of the patient should be monitored for at least 3 months after Zolgensma administration.
Some vaccines are contraindicated in patients receiving a substantially immunosuppressive dose of steroids. Therefore, caregivers should consult with their health care professional to determine if adjustments to the patient's schedule for vaccination are necessary to allow concomitant administration of corticosteroids, the FDA said.
The FDA has granted the Zolgensma application an expedited procedure, innovative treatment and prioritization designations, as well as orphan drug designation. The FDA has also granted the manufacturer a priority review voucher for rare pediatric diseases as part of a program to encourage the development of new drugs and biologics for the prevention and treatment of some rare pediatric diseases.
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