ZURICH / NEW YORK – The Swiss drug maker Novartis received US approval for its Zolgensma gene therapy on Friday for spinal muscular atrophy (SMA), the leading cause of genetic death among infants, and a one-time treatment at a record low price $ 2.125 million.
The Food and Drug Administration approved Zolgensma for children under two with SMA. including those who do not show symptoms yet. The approval is for babies with the most deadly form of hereditary disease, as well as for babies with types, which can later debilitating symptoms.
Dr. Emmanuelle Tiongson, a pediatric neurologist at the Children's Hospital in Los Angeles, who provided patients with Zolgensma as part of an extended access program. "The task now is to negotiate with the insurers that this represents a long-term saving."
Novartis executives defended the award, stating that a one-time treatment would be more valuable than expensive long-term treatments that cost hundreds of thousands of dollars a year.
Novartis sparked a debate over the value of gene therapy last year, estimating that treatment at up to $ 5 million per patient would be cost-effective. An April review by an independent US group, the Institute for Clinical and Economic Review (ICER), concluded that the valuation made by Novartis for Zolgensma was too high Novartis announced that it offers the possibility to health insurers The payment of installments for Zolgensma and reimbursements in the event of treatment is inoperative and grants discounts in advance to payer who commit to standardized cover conditions. Vas Narasimhan, Chief Executive of Novartis, is keen on Zolgensma and describes this as a cure for SMA if it is delivered shortly after birth.
However, the data showing durability only last for about five years. The therapy uses a virus to provide a normal copy of the SMN1
Novartis expects approval in Europe and Japan later this year. Zolgensma will compete with Spinraza of Biogen Inc., the first approved treatment for SMA.
The disease often causes paralysis, difficulty in breathing and death within a few months in the most severe type I infants. SMA affects approximately one in 10,000 live births, with 50 to 70 percent suffering from a type I disease.
Spinraza, which was approved at the end of 2016, must be infused every four months. The list price of $ 750,000 for the first year and $ 375,000 a year was also considered excessive by ICER.
Some neurologists consider gene therapy to be the preferred treatment for newborns with severe SMA, while recognizing that families may be waiting for long-term stays. Safety and efficacy data for Zolgensma. Novartis examines whether the death of a seriously ill child treated with Zolgensma is related to the therapy.
"Most families will want to do gene therapy because it avoids the frequent inflammation of the spine," Dr. Russell Butterfield from the University of Utah in Salt Lake City. Butterfield has received payments from Biogen for the consultation.
The FDA claimed to have approved Zolgensma based on clinical trials in 36 patients, ages 2 weeks to 8 months. According to the agency, patients treated with Zolgensma showed significant improvement in motor development milestones such as head control and sitting ability.
The most common side effects with Zolgensma are increased liver enzymes and vomiting. The FDA requires Zolgensma to warn that acute severe liver damage may occur.
With additional ongoing studies, Novartis has already treated more than 150 patients with Zolgensma, acquired last year with the purchase of AveXis worth $ 8.7 billion.
Wall Street analysts forecast $ 2 billion in sales by 2022, according to a refine survey. Spinraza generated $ 1.7 billion in revenue last year and is projected to reach $ 2.2 billion in 2022 Dollar rise. Roche is developing Risdiplam, a prescription drug, in case it's being applied for later this year
Novartis, Biogen and Roche as well as patient advocates and neurologists say babies are being treated with SMA before the onset of symptoms will have the best chance of nearly normal development. They are committed to making SMA neonatal screening the norm in every market.
"Babies (with SMA) lose motor neurons from the day they are born, and the ability to treat them as early as possible is the best way to treat them." David Lennon, who heads the AveXis unit at Novartis, said in an interview recently. Laurent Servais, a pediatric neurologist in Liege, Belgium, described any delay in performing neonatal SMA screening as "completely unethical".
Servais helped monitor a screening pilot program sponsored by the three companies. In southern Belgium, 60,000 newborns are being studied each year, half of the country's total births. Taiwan has also begun to test babies for SMA.
However, widespread adoption is still a long way off. In the US, only six states have started an active and routine SMA neonatal screening since the federal government recommended it in 2018. Patient representatives estimate that it could take until 2022 for tests to be conducted nationwide.
In Europe, this may be the case even slower.
England advised against newborn screening in February and will not consider it again until 2021, a Public Health England spokesperson told Reuters. SMA advocates in Germany do not expect screening measures until the end of 2021.
"It's amazing," said Inge Schwersenz of the German Society for Neuromuscular Diseases. "But we can not do anything to speed it up."