Novartis Sets $4–5 Million Price Tag for One-Time Gene Therapy AVXS-101 Ahead of Expected 2019 Launch

After pricing Kymriah, the world’s first approved CAR-T therapy, at $475,000, Novartis has priced its upcoming new gene therapy at $4 million to $5 million (approximately at least RMB 28 million).

It is understood that the aforementioned novel gene therapy, named AVXS-101, is primarily indicated for the treatment of spinal muscular atrophy (SMA) and represents AveXis’s flagship gene therapy product. In April this year, Novartis acquired AveXis for $8.7 billion through its indirect wholly-owned subsidiary, Novartis AM Merger Corporation, thereby bringing AVXS-101 into its portfolio. In mid-last month, AveXis announced the latest progress on the AVXS-101 therapy and stated that it had submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its use in Type 1 SMA. Similar applications have also been submitted to regulatory authorities in the European Union and Japan. It is expected that all three regulatory agencies will render their decisions by mid-2019, which means that AVXS-101 could be approved for marketing in 2019, becoming the second gene therapy approved for the treatment of SMA.The first approved SAM gene therapy is Spinraza）。

Meanwhile, it is worth noting that if Avexis is approved, AVXS-101 will become the “first-in-class” one-time gene replacement therapy, addressing the root genetic cause of SMA by effectively replacing the defective SMN1 gene. Previously, the U.S. FDA had granted AVXS-101 orphan drug designation and breakthrough therapy designation.

According to Reuters, Novartis CEO Vas Narasimhan revealed on Monday that although Novartis has not yet officially announced the market price for this gene therapy, it may set the price at $4 million or more. Specific pricing is currently under negotiation with insurance companies and relevant government health insurance agencies in the United States, Europe, and Japan. Meanwhile, AveXis President Dave Lennon also stated, “$4 million is a substantial sum, but we believe this represents a cost-effective perspective. Other studies have shown that our treatment is cost-effective within the range of $4 million to $5 million. An important context is that we are considering how to evaluate the therapeutic value of AVXS-101.”

What kind of therapy is this, that it has been assigned such a high price?

Let us begin by discussing spinal muscular atrophy (SMA), the condition treated by AveXis. SMA is a hereditary neurodegenerative disease caused by defects in the single-gene survival motor neuron 1 (SMN1) gene. It directly leads to muscle weakness and atrophy of motor neurons, potentially resulting in paralysis. The disease severely affects respiratory muscles, compromising respiratory function. Based on the age of onset and severity, SMA is classified into four types: Type I, Type II, Type III, and Type IV. Approximately 60% of SMA patients have Type I SMA, the most severe form. Symptoms appear between birth and six months of age and are characterized by profound muscle weakness, including the inability to hold up the head, breathe normally, cough, or swallow, with patients able only to sit with support. SMA has become the leading genetic cause of infant mortality; 90% of infants with this disease do not survive beyond two years of age or require permanent dependence on mechanical ventilation for survival. It is estimated that one in every 6,000 to 10,000 children is affected by some form of SMA.

In previous clinical trials for spinal muscular atrophy (SMA), AVXS-101 consistently demonstrated positive outcomes. In an early Phase I clinical study of AVXS-101, all 15 enrolled infants with Type 1 SMA achieved a 100% event-free survival rate and maintained normal respiratory function by the age of 20 months, whereas the probability of such outcomes without treatment was only 8%. Among the 12 infants who received the optimal dose of AVXS-101 (the remaining three received a lower dose), the CHOP INTEND scores increased by 9.8 points one month after administration and by 15.4 points three months after administration, up to the end of the Phase I trial.

Meanwhile, AVXS-101 is currently being evaluated in the pivotal clinical trial Str1ve, which has enrolled 15 infants with Type 1 SMA under 6 months of age, primarily assessing event-free survival and the proportion of patients maintaining independent respiration.

Industry insiders estimate that once AVXS-101 is approved for market launch, it will complement Kymriah to become a strong revenue growth driver for Novartis. According to forecasts by the evaluation firm EvaluatePharma, global sales of AVXS-101 are expected to reach $1.339 billion in 2024.