Novartis, a $ 2 million gene therapy for the treatment of rare diseases, is the world's most expensive drug

ZURICH / NEW YORK – Swiss drug maker Novartis on Friday received US approval for its Zolgensma gene therapy against spinal muscular atrophy (SMA), the leading genetic cause of death in infants, and assessed single-drug treatment record price of $ 2.125 million.

The Food and Drug Administration has approved Zolgensma for children under two years of age with ADS, including those who do not have symptoms yet. Approval concerns babies with the most lethal form of the hereditary disease as well as those with types where debilitating symptoms may occur later.

"It's potentially a new standard of care for babies with the most severe form of ADS," said Dr. Emmanuelle Tiongson, pediatric neurologist at Los Angeles Children's Hospital. Angeles, who provided Zolgensma to patients as part of an expanded program of access. "The job now is to negotiate with insurers to save money in the long run."

Novartis executives defended the award, saying that a single treatment was more valuable than an expensive long-term treatment costing several hundred thousand dollars a year.

Novartis launched a debate on the value of gene therapy last year, estimating that its treatment would be profitable at $ 5 million per patient. An April review by an independent US group, the Institute for Clinical and Economic Review (ICER), found that Novartis' estimate of Zolgensma's value was excessive.

However, on Friday, ICER said that based on additional clinical data from Novartis, the FDA wide label and its introductory price, ICER believed the drug was in the upper range of its profitability range.

Novartis indicated that it offers health insurers the possibility of making installment payments for Zolgensma, as well as refunds if the treatment was not working and initial discounts for payers committing to standardized coverage conditions.

Novartis chief executive Vas Narasimhan has a lot to say about Zolgensma, which he describes as a virtual cure for the SMA if it is delivered shortly after birth. But the data proving its durability extend only about five years. The therapy uses a virus to provide a normal copy of the SMN1 gene to babies born with a defective gene. It is delivered by infusion.

A RIVAL AT BIOGEN

Novartis is awaiting European and Japanese approval later this year. Zolgensma will compete with Spinraza of Biogen Inc, the first approved treatment for ADS.

The disease often results in paralysis, breathing difficulties and death within a few months for infants born with the most severe form of type I. SMA affects about one in 10,000 births, with 50 to 70% of type I.

Spinraza, approved late 2016, requires infusion into the spinal canal every four months. Its catalog price of $ 750,000 for the first year and $ 375,000 thereafter was also deemed excessive by ICER.

Some neurologists consider that gene therapy is becoming the treatment of choice for newborns with severe AS, while recognizing that families may choose to wait for the data relating to the condition. safety and long-term effectiveness of Zolgensma. Novartis is investigating whether the death of a critically ill baby being treated with Zolgensma was treatment-related.

"Most families will want to do gene therapy because it avoids frequent spinal taps," said Dr. Russell Butterfield of the University of Utah in Salt Lake City. Butterfield has received payments from Biogen for consultation.

The FDA has approved Zolgensma on the basis of clinical trials of 36 patients aged 2 weeks to 8 months. According to the agency, patients treated with Zolgensma showed a significant improvement in developmental milestones such as head control and the ability to sit down.

The most common side effects of Zolgensma are elevated liver enzymes and vomiting. The FDA is asking Zolgensma's label to include a warning stating that a serious and serious liver injury may occur.

With additional studies underway, Novartis said it has so far treated more than 150 patients with Zolgensma, acquired when AveXis was purchased for $ 8.7 billion last year.

Wall Street analysts expect a turnover of $ 2 billion by 2022, according to a survey of Refinitiv. Spinraza sales reached $ 1.7 billion last year and are expected to reach $ 2.2 billion in 2022. Roche is developing the oral drug risdiplam and is considering approving it later this year .

A PUSH BUTTON FOR SCREENING

Novartis, Biogen and Roche, as well as patient rights advocates and neurologists, say that babies with AMS who receive treatment before the symptoms occur have the best chance of almost normal development. They are pushing to make the SMA screening standard for newborns in all markets.

"Babies (with ADS) lose motor neurons right from birth, so the ability to treat them as early as possible is the best way to get the most out of the therapy," said David Lennon, head of the hospital. AveXis unit of Novartis, said in a recent interview.

Dr. Laurent Servais, a neurologist for children in Liege, Belgium, has described as "completely unethical" any delay in the implementation of screening for AMS in the newborn.

Servais helped oversee a pilot screening program sponsored by the three companies. Southern Belgium is currently screening 60,000 newborns annually, half of all births. Taiwan has also started testing babies for the SMA.

But widespread adoption still has a long way to go. In the United States, only six states have begun active and routine screening of ADM infants since the federal government recommended it in 2018. Patient advocates believe that it may be necessary to until 2022 for the test to be implemented nationwide.

In Europe, it can be even slower.

England recommended against neonatal screening in February and will not review it further until 2021, a spokesman for Public Health England told Reuters. Defenders of the SMA in Germany do not expect action on screening before the end of 2021.

"It's amazing," said Inge Schwersenz, of the German Society of Neuromuscular Diseases. "But we can not do anything to speed things up."