Landmark Month Sees Second Gene Therapy Approved and Other Wins

RxC International Gene Therapy Newsletter

October was a landmark month for gene therapies with the approval of the second-ever gene therapy in the US, Yescarta (axicabtagene ciloleucel) from Kite and Gilead.  The prior approval of Novartis’ Kymriah is more proof that gene therapies are set to shake up the pharma industry.  Gene therapies saw other wins as Luxturna, Spark’s treatment for Leber congenital amaurosis, received the FDA’s recommendation for approval and Bluebird Bio’s interim data report for its gene therapy Lenti-D to treat cerebral adrenoleukodystrophy showed promising results.

That’s not to say it was all smooth sailing.  Pricing remains forefront in the discussion of gene therapies’ value and potential to “break the bank.”  And not all therapies are succeeding as Alnylam suspended its RNAi fitursiran trial after a patient death.

RxC International will continue to closely monitor gene therapy developments to provide readers with news and insights into this ever-evolving market.


Pricing Debate Intensifies with Approval of First Gene Therapies

The recent approvals of the first two gene therapies in the United States have intensified the debates over pricing and the financial burden these treatments may have on the healthcare system.

While gene therapies are emerging onto the commercial market, the debate surrounding affordability is not new.  Dating back to the era of Lipitor and other blockbuster PCP-driven drugs to the expansion of the oncology market to the more recent emergence of multiple rare disease treatments, the question has always been the same: “How can we afford them?”  The real question, however, is not whether we a society can afford gene therapy treatments, but whether we can afford not to pursue treatments that extend or improve people’s lives.


Gene Therapy Pricing and Target Patient Population


The article offers some important insight into the pricing debate, but at the same time makes the fundamental mistake of grouping all gene therapies into a single category.  Gene therapies are far from a homogeneous group so must be thoughtfully segmented to fully understand the different dynamics at play for each product.

The chart seems to purport an overall correlation between pricing and target patient population, however it doesn’t take into account the basic HEOR measure of cost per year of life saved.  While gene therapies targeting rare diseases may have a smaller patient pools, they often target childhood diseases with the potential to add decades to each patient’s life. Oncology is a very different dynamic.


Harvard Scientists Reveal New Gene Editor

Using CRISPR-Cas9 technology, scientists can now change a single DNA letter.  This advancement expands the capabilities of the first base editor, created last year, by converting additional bases.

While CRISPR continues to make strides for gene therapy advancement, it is still a long way from creating commercial therapies.


Research Updates

Second Gene Therapy Treatment Approved

The October 18th approval of Yescarta (axicabtagene ciloleucel), a CAR-T treatment for adults with relapsed or refractory large B-cell lymphoma, places Kite and Gilead in the new market of approved US gene therapies. Novartis’ Kymriah, the first gene therapy to gain approval in the US, is also a CAR-T therapy.  While currently aimed at different oncology segments, the two CAR-T treatments could become competitors as both brands seek to expand product labels. 


Fitusiran's Recent Failure Opens Door for Additional Therapies

The September suspension of trials for RNAi fitursiran following the death of a patient in one of the studies has opened the door for two competing gene therapies that offer long-term benefit from one administration.


Gene Therapy Receives Unanimous Recommendation for Approval

Luxturna, a treatment for a hereditary form of blindness, received recommendation for approval from an FDA advisory committee.  Spark Therapeutics, the treatment’s developer, is expected to receive a decision from the FDA by January.


Encouraging Results for Gene Therapy Treatment of Fatal Neurodegenerative Disease

Interim data for Bluebird Bio’s gene therapy treatment Lenti-D for boys suffering from cerebral adrenoleukodystrophy (CALD) showed encouraging results.  Of the 17 boys who received treatment as part of Bluebird’s trial, 15 were alive and stable after 24 months. "The clinical experience with Lenti-D demonstrates the potential for gene therapy to benefit patients with CALD," said David Davidson, MD, chief medical officer of Bluebird Bio.  The company is currently considering its plans for FDA approval.

About RxC International

Understanding the subtleties of the gene therapies is critical for bringing these treatments to market. RxC International has extensive experience successfully commercializing and launching new drug products, developing innovative solutions, realizing a product’s best potential, and working across organizations to achieve common goals.

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About the Author

Richard Tinsley is a Strategy Partner at RxC International with over 20 years of life sciences consulting experience. He is a recognized leader in strategy, commercialization, life cycle management, and sophisticated forecast modeling. Rich has deep expertise in specialty markets such as rare diseases, HIV, oncology, neurology, and diagnostics.