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The US Food and Drug Administration on Friday approved Zolgensma (onasemnogene abeparvovec-xioi), the first approved gene therapy for the treatment of children under two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a major genetic cause of infant mortality.
The Zolgensma label, a unique treatment, imposes an age limit less restrictive than that anticipated by badysts. It is important to note that this will also allow Zolgensma to capture all patients with type 1 and type 2 AMS without requiring neonatal screening – patients with type 1 develop symptoms by 6 months of age, and Type 2 patients usually develop symptoms between 7 and 18 months. Mani Foroohar, Analyst at SVB Leerink Research.
Following the FDA's announcement, gene therapy developer AveXis, a subsidiary of the Swiss pharmaceutical giant Novartis (NOVN: VX), has announced innovative access programs to Zolgensma for the treatment of pediatric patients less than two years of SMA with bi-allelic mutations in survival motor neuron 1 (SMN1) the gene.
AveXis, which Novartis acquired last year under a $ 8.7 billion contract, works closely with payers to offer payment options for up to five years and based on results for up to five years, as well as a patient-friendly program. and access. Novartis shares rose nearly 4% on Friday late afternoon.
Jeffries badysts predict that Zolgensma's sales will reach $ 2.6 billion, exceeding estimates of the $ 1.9 billion consensus sales peak.
Cost of therapy
The current cost of chronic treatment over 10 years, administered throughout the patient's life, can often exceed $ 4 million during the first 10 years of a young child's life. In addition, this therapy stops working if treatment is stopped. Zolgensma is expected to reduce costs in the health system compared to chronic treatment of ADS treatment and care.
The wholesale acquisition cost set by the company for Zolgensma is $ 2.125 million and is raised at:
- 50% of the current cost of chronic AMS treatment over 10 years (estimated at $ 4.1 million);
- 50% less than the 10-year treatment cost for very rare genetic diseases in children (estimated at between $ 4.4 and $ 5.7 million) and
- 50% below the cost-effectiveness threshold of the ultra-rare ICER disease; The price of Zolgensma is about 250,000 USD per year adjusted life based on quality (QALY).
The list price is slightly lower than that anticipated by Leerink's Mani Foroohar, who stated that "Zolgensma's catalog price is $ 2.125 million per patient, paid over five years at an annualized cost of $ 425,000 per year. . If a conservative gross / net adjustment of 90% were badumed, the announced price would predict a net price of $ 1.9125 million, which is slightly lower than our $ 2 million net price estimate / dose , although we note that Zolgensma's actual net price could be lower due to Medicaid's mandatory discounts, and some patients fail over time to comply with the outcome measures defined in VBA payers (value-based agreement). "
"Historical Advancement for ADM Treatment"
"Zolgensma represents a historic breakthrough in the treatment of ADS and unique gene therapy, and aims to ensure broad patient access to this transformational medicine and to share value with the health system," said Dr. Vas Narasimhan. Chief Executive Officer of Novartis. "We used value-based pricing systems to set Zolgensma at a price that is about 50% lower than several benchmarks, including the current cost of chronic AMS treatment over 10 years. In addition, the price of Zolgensma should be in the range of traditional costs ICER when updating its full indications.We believe that by adopting this responsible approach, we will help patients to leverage this transformative medical innovation and generate significant cost savings for the system over time, "he explained.
"Today's approval marks a new step in the transformative power of gene and cell therapies to treat a wide range of diseases," said Dr. Ned Sharpless, Acting Commissioner of the FDA. "With each new approval, we find that this exciting scientific field continues to move beyond the design phase to become a reality. The potential of gene therapy products to change the lives of these patients who may have faced a life-threatening illness or, worse, death, gives hope in the future, "he added.
"Zolgensma is radically transforming the lives of families affected by this devastating disease and, given the new presymptomatic population efficacy data, the price announced today is within the upper limit of the reference range of the disease. ICER price, "said Dr. Steven Pearson, chair of the Profit Monitoring of the Institute for Economic and Clinical Review (ICER). "The insurers were going to cover Zolgensma, whatever the price, and Novartis has publicly talked about pricing around $ 5 million. For patients and the entire healthcare system, Novartis has chosen to set the price of Zolgensma at a level that better matches the benefits for these children and their families, "he added.
This updated reference price based on the Zolgensma value badumes that the efficacy of the treatment will be sustainable and that the United States is broadly and rapidly adopting the recommendation to add systematic screening of AMS for routine newborn screening. Once additional data become available, the ICER has announced the possibility of updating the new evidence for Zolgensma and nusinersen, the previously marketed Biogen SMA Spinraza (Nasdaq: BIIB) treatment.
At the present time, excluding any markup, Biogen's Spinraza list price in the United States is $ 750,000 for the first year and $ 375,000 annually thereafter. Last year, sales totaled $ 1.7 billion.
Outside of the United States, Zolgensma has the PRIME designation (priority drugs) in Europe and is currently under review as part of the expedited review procedure. It has also accelerated Sakigake designation in Japan. At the same time, Roche (ROG: SIX) is developing a daily oral treatment for ADM, called risdiplam, which, according to its forecasts, is expected to obtain regulatory approval this year and be marketed in 2020.
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