Question: How Many Patients Are Treated With Zolgensma?

Who invented Zolgensma?

Zolgensma was developed by AveXis, a Chicago-headquartered biotech that Novartis bought in 2018 for $8.7bn.

AveXis’ intellectual property holdings and licensing agreements are key according to Althoff, who cites the firm’s deal with Rockville, Maryland-based Regenxbio as an example..

Is Zolgensma a gene therapy?

Zolgensma (onasemnogene abeparvovec-xioi), marketed by Novartis Gene Therapies, is an FDA-approved therapy to treat SMA. It is a type of treatment referred to as gene therapy or gene replacement therapy.

What is the most expensive prescription drug?

The 20 Most Expensive Drugs in the U.S.DrugList Price1Myalept$71,3062Mavenclad$56,9543Ravicti$55,3414Actimmune$52,77716 more rows•Aug 11, 2020

Are there any approved gene therapies?

The U.S. Food and Drug Administration (FDA) has approved only a limited number of gene therapy products for sale in the United States. Hundreds of research studies (clinical trials) are under way to test gene therapy as a treatment for genetic conditions, cancer, and HIV/AIDS.

How common is the SMA gene?

It is a one of the most common genetic conditions affecting children. It is estimated that one in every 6,000 to 10,000 babies worldwide is born with SMA. In more than 95 percent of cases, SMA is caused by inadequate production of a protein called survival motor neuron (SMN) protein that is essential to motor neurons.

Does insurance cover Zolgensma?

More than 20 private health-insurance plans and four Medicaid plans have set up coverage policies for Zolgensma since the FDA approval, according to Novartis. The drugmaker also noted that it was testing Zolgensma in older patients with spinal muscular atrophy, in hopes of getting the therapy approved for them.

Is Zolgensma a biologic?

Onasemnogene abeparvovec-xioi is an original biologic product, with the proprietary name of ZOLGENSMA. … ZOLGENSMA is designed to deliver a normal copy of the gene encoding the SMN protein in patients with SMA.

Where is Zolgensma manufactured?

Novartis makes Zolgensma at a site in Libertyville, Illinois, which it acquired along with the drug’s developer AveXis in 2018.

How much does SMA treatment cost?

A $2.1 million treatment for SMA, though, is no more ludicrous than paying more than $1,000 a month to rent a portable ventilator, especially when that one treatment will likely eliminate the need for the ventilator and many other costs associated with SMA care.

How long does Zolgensma last?

New data from the START long-term follow-up study continue to demonstrate the durability of a single, one-time dose of Zolgensma in patients now up to five years post-dosing and some patients more than five years of age.

Why is gene therapy so expensive?

The main reason gene therapy is so expensive, however, may be the paradigm used in the price-setting strategy. The cost of production is weighed against the value of a life saved or the improved quality of life over a specified timeframe.

How does Zolgensma work?

As a gene therapy, ZOLGENSMA® (onasemnogene abeparvovec-xioi) is designed to target the genetic root cause of spinal muscular atrophy (SMA) by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of a human SMN gene. ZOLGENSMA does not change or become a part of the child’s DNA.

Is SMA rare?

Spinal muscular atrophy (SMA) is a progressive genetic disorder that affects the nervous system and muscles, and is a very rare disease at that, found in an estimated 1 in every 6,000 to 1 in every 10,000 people.

How often is Zolgensma given?

Troponin-I weekly for the first month, and then monthly for the second and third months, until troponin-I level returns to baseline. ZOLGENSMA is a suspension for intravenous infusion. ZOLGENSMA is provided in a kit containing 2 to 9 vials. Vials are provided in 2 fill volumes: 5.5 mL or 8.3 mL.

Why Zolgensma is so expensive?

The reason Zolgensma is so expensive is because that is the price Novartis has decided it is worth because it “dramatically transforms the lives of families affected by this devastating disease” and the claimed cost of bringing new drugs to market. But this price is not without controversy.

How much does Zolgensma cost?

At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever. Zolgensma, a new drug approved by the FDA Friday, costs more than $2.1 million. It’s made by AveXis, a drugmaker owned by pharmaceutical giant Novartis.

What is the most expensive antibiotic?

The cephalosporins are among the most expensive antibiotics in use today; thus, use of these expensive agents must be justified by lower toxicity, greater efficacy, or both in comparison with drugs of more reasonable cost.