Novartis, after pricing the world's first approved CAR-T therapy, Kymriah, for $475,000, will price $4 million to $5 million (approximately at least 28 million yuan) for its new gene therapy. .
It is understood that the above new gene therapy is called AVXS-101, mainly used for the treatment of spinal muscular atrophy (SMA). It is a heavy gene therapy product of AveXis. In April this year, Novartis passed its indirect wholly-owned subsidiary. Novartis AM Merger Corporation acquired AveXis for $8.7 billion, which naturally brought AVXS-101 together. In the middle of last month, Avexis announced the latest developments in AVXS-101 therapy and said it has submitted its use to the US FDA. For applications for Type 1 SMA Biologics Licensing (BLA), similar applications have been submitted to regulatory authorities in the European Union and Japan. It is expected that by the middle of 2019, all three regulatory agencies will make a decision, which also means AVXS- 101 or will be approved for marketing in 2019, becoming the second gene therapy for the treatment of SMA (the first approved SAM gene therapy is Spinraza).
At the same time, it is worth mentioning that if Avexis is approved, AVXS-101 will become a “first-in-class†one-time gene replacement therapy, which will solve the underlying genetic cause of SMA by effectively replacing the defective SMN1 gene. Previously, the US FDA has granted AVXS-101 orphan drug qualification and breakthrough therapy approval.
According to Reuters, Novartis CEO Vas Narasimhan revealed on Monday that Novartis has not officially announced the market price of the gene therapy, but may set the price at $4 million or more. , European and Japanese medical insurance companies and related government medical insurance institutions are negotiating. At the same time, AveXis president Dave Lennon also said: $4 million is a substantial amount of money, but we believe this is a cost-effective view, through other studies, we have only in the range of 4 million to 5 million US dollars Cost-effective, and an important background is how we are considering how to evaluate the therapeutic value of AVXS-101. "
What kind of therapy is this, will it be set to such a high price?
First of all, AveXis treatment of SMA, a hereditary neurodegenerative disease caused by a single gene motor neuron survival gene SMN1 deficiency, will directly lead to muscle weakness and motor neuron atrophy and even paralysis, a serious impact Respiratory muscles affect respiratory function. Depending on the time and severity of the disease, SMA can be divided into type 1, type 2, type III and type IV. About 60% of patients with SMA are type 1 SMA, which is also the most serious one. The type, which occurs between the child's birth and 6 months, is characterized by severe muscle weakness, including failure to look up, unable to breathe normally, coughing or swallowing, and can only sit upright. SMA has become the number one genetic cause of infant death, and 90% of infants with this disease cannot survive 2 years of age or need to rely on a ventilator for permanent survival. It is estimated that one out of every 6,000-10,000 children is affected by some form of SMA.
In the previous clinical trials for SMA, AVXS-101 received positive data. In a clinical phase I study in the early AVXS-101, 15 infants with type 1 SMA were enrolled as infants. At 20 months of age, the event-free survival rate is 100% and can be breathed normally, compared to 8% when not receiving treatment. Among the 12 infants given the appropriate dose of AVXS-101, the end of the clinical phase I trial ended. For the 12 patients enrolled in the recommended dose (the other 3 were low doses), the Chop-Intend score increased by 9 after 1 month of administration. .8, an increase of 15.4 after 3 months.
At the same time, AVXS-101 is still in the ongoing critical clinical study Str1ve, enrolling 15 patients with type 1 SMA infants younger than 6 months, mainly to assess the event-free survival rate and spontaneous breathing rate of infants.
According to industry insiders, once approved, AVXS-101 will be combined with Kymriah to form a strong growth point for Novartis's revenue. According to the evaluation agency EvaluatePharma, the global sales of AVXS-101 will reach 13.39 in 2024. One hundred million U.S. dollars.
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