Foreword:
In the ever-evolving landscape of gene therapies, the success stories of pioneering treatments like Kymriah from Novartis serve as beacons of hope for patients and industry enthusiasts alike. As we eagerly anticipate the potential FDA approval of CRISPR Therapeutics and BlueBird Bio’s groundbreaking gene therapies, let’s take a moment to delve into the transformative journey that led to Kymriah’s approval and subsequent market performance. By understanding the strategic alliances, regulatory milestones, and commercialization efforts that shaped Kymriah’s trajectory, we can begin to visualize the future paths of Exa-cel and Lovo-cel.
Formation of Innovative Partnership (2012):
The journey of Kymriah began in 2012 when the University of Pennsylvania (Penn) forged a groundbreaking collaboration with Novartis. This innovative partnership laid the foundation for a transformative alliance program focused on the development of CAR-T cell therapy, eventually giving birth to Kymriah.
Strategic Alliance Structure (2012-2013):
The Partnership for Cellular Therapeutics (PCI) actively shaped and negotiated the alliance deal, setting the stage for a unique agreement structure. The focus was on facilitating basic CAR-T research, technological development, and the seamless clinical translation of Kymriah. A robust system for sharing and tracking materials between Penn and alliance partners was established during this period.
Program Expansion and Management Flexibility (2013-2020):
Over the next eight years, PCI dynamically managed the alliance, adapting to evolving research needs. Collaborations expanded with other academic institutions, contract manufacturing partners were engaged, and the PCI Alliance team grew. The alliance successfully met increasingly complex obligations, including manufacturing engagement in Phase II multi-site clinical studies in 2014.
Restructuring for Research Alignment (2013-2020):
PCI Alliance played a pivotal role in restructuring the alliance relationship to align with the evolving research program. Close collaboration with Penn researchers and Novartis ensured that the alliance consistently supported the direction of CAR-T research.
FDA Approval of Kymriah (2017):
A significant milestone was reached in 2017 when the FDA granted approval to Kymriah, with the distinction of receiving the first unanimous recommendation for approval from the Oncologic Drugs Advisory Committee.
Facilitation of Commercialization Transition (2017-2020):
PCI Alliances worked diligently to create mechanisms facilitating the smooth transition of the CAR-T program from Penn’s translational research laboratories to Novartis’ commercialization teams and facilities.
The Path to Commercialization:
With Kymriah’s approval secured, Novartis embarked on the challenging journey of bringing this revolutionary therapy to the market. Recently, Novartis announced production pacts in China, adding to existing agreements in France, Germany, and Switzerland. The company is actively expanding its manufacturing capabilities, building a Kymriah factory in Switzerland.
The intricate process of manufacturing CD19 targeting autologous therapy involves harvesting, modifying, and re-infusing patient cells. Novartis, in its commitment to global accessibility, aims to lead distribution, commercialization, and approval efforts in China, though the status of Kymriah’s progress with Chinese drug regulators remains undisclosed.
Price, Cost Speculation, and Market Performance:
According to Novartis, the Kymriah cancer therapy is priced at $475,000 for one treatment course. Before its launch, analysts speculated on the cost, with some pegging it at $200,000 per dose, while British authorities suggested $650,000 could be justified if it added years to children’s lives. The Institute for Clinical and Economic Review (ICER) approved the price tag after conducting a thorough analysis.
Here’s how Kymriah’s sales have unfolded:
- 2019: $278 million USD
- 2020: $474 million USD (68% growth)
- 2021: $587 million USD
- 2022: $536 million USD
- 2023 (first 3 quarters): $388 million USD
These figures provide a solid foundation for visualizing the potential trajectories of upcoming gene therapies like Exa-cel and Lovo-cel.
In conclusion, the journey of Kymriah serves as a compelling narrative of collaboration, innovation, regulatory milestones, and successful commercialization. As we await the future endeavors of CRISPR Therapeutics and BlueBird Bio, the lessons learned from Kymriah’s path can guide our expectations and aspirations for the next wave of transformative gene therapies.
Link to video: https://youtu.be/kPEAMTLbH10
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