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www.psgconsults.com/blog/fda-delayed-approval-of-gene-therapy-drug

It was expected to be the most expensive drug ever approved by the Food and Drug Administration (FDA), at an estimated price tag of as much as $3 million. However, Roctavian, the gene therapy for hemophilia A, failed to meet the FDA standards for approval on August 19, 2020.

geneticliteracyproject.org/2020/02/12/900-gene-therapy-drugs-are-in-the...

To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency anticipates many more approvals in the coming years, as evidenced ...

www.pharmamanufacturing.com/articles/2019/the-rise-of-gene-therapies

But now, when a gene therapy comes onto the market, it becomes the world’s new “most expensive drug” (the crown was most recently passed to Novartis’ Zolgensma which treats an inherited form of spinal muscular atrophy for about $2.1 million).

www.washingtonpost.com/business/economy/the-fda-approves-a-gene-therapy-that...

The Food and Drug Administration on Friday approved the most expensive drug in the world, a $2.1 million one-time gene therapy for treatment of a rare disease that afflicts infants.

clarkstonconsulting.com/insights/fda-gene-therapy-guidances

On January 28, 2020, the highly anticipated final FDA gene therapy guidances were released. In total, 7 guidance documents were issued, focusing on gene therapy topics for organ drugs, specific diseases, Chemistry, Manufacturing, and Controls (CMC) for Investigational New Drugs (IND), patient follow-up after drug administration, and testing on retroviral vector-based therapies.

www.cnbc.com/2019/05/24/fda-approves-novartis-2-million-spinal-muscular...

The Food and Drug Administration on Friday approved Novartis' $2.1 million gene therapy for spinal muscular atrophy — making it the world's most expensive drug. The therapy, Zolgensma, is a one ...

www.risingtidebio.com/what-is-gene-therapy-uses

It has finally happened, we now have a gene therapy approved in the US with a price-tag over 1 million…actually, well over 1 million. Zolgensma, a gene therapy for spinal muscular atrophy, was approved by the FDA in 2019. Novartis has priced this therapy at $2.1 million per patient. The science behind this new drug is ground-breaking.

medlineplus.gov/genetics/understanding/therapy/availability

Gene therapy is currently available primarily in a research setting. 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.

www.mckesson.com/biopharma/cell-gene-therapy

Build a Successful Commercialization Strategy for Your Cell and Gene Therapy Cell and gene therapies have transformed the traditional drug commercialization model with one that meets the individual needs of providers, patients and payers. We can help you create an innovative strategy tailored to your therapy's needs.

medlineplus.gov/genetics/understanding/therapy/genetherapy

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including: