Gene Therapy Setback: Sangamo’s Stock Plummets 50% After Pfizer Ends Partnership
A Major Blow to Haemophilia A Treatment
Sangamo Therapeutics’ stock has taken a drastic hit, plummeting 50% in premarket trading, following Pfizer’s decision to terminate their partnership to co-develop a gene therapy for haemophilia A. This sudden move has left Sangamo regaining the development and commercialization rights to giroctocogene fitelparvovec, an investigational gene therapy for adults with moderately severe to severe haemophilia A.
Positive Results Marred by Partnership Termination
Despite the promising results from the Phase III AFFINE clinical trial, which demonstrated a significant reduction in the total annualized bleeding rate compared to routine Factor VIII replacement prophylaxis, Pfizer has opted not to proceed with the biologics license application and marketing authorization application filings. This decision comes as a surprise, given the therapy’s potential to be life-changing for haemophilia A patients.
The Challenges of Gene Therapy Development
The development of gene therapies for rare diseases is a complex and resource-intensive process, requiring advanced technologies, large-scale clinical trials, and navigation of complex regulatory pathways. This has led several pharmaceutical companies to reevaluate or halt gene therapy programs due to high costs and questions about commercial viability. Gene therapies, especially for conditions with small patient populations like haemophilia A, pose unique financial challenges.
The Cost Conundrum
While gene therapies can potentially provide long-term or curative outcomes, their high development costs and production complexities result in significant price tags. For instance, BioMarin’s FDA-approved haemophilia A gene therapy, Roctavian, launched in 2023 with a $2.9m price point, sparking ongoing debate about the cost-effectiveness of such therapies.
Sangamo’s Next Steps
Sangamo plans to explore options to advance the program, including seeking a new collaboration partner. CEO Sandy Macrae expressed disappointment at Pfizer’s decision but acknowledged Pfizer’s role in advancing the program to this stage. Macrae remains optimistic about the therapy’s potential, stating that it is well-positioned for regulatory submissions and commercialization.
A Setback for Haemophilia A Patients
Pfizer’s decision to terminate the partnership has significant implications for haemophilia A patients, who were hoping for a new treatment option. The company’s decision to halt the program due to slow uptake and limited interest in another gene therapy option for the specified patient population raises concerns about the future of gene therapy development for rare diseases.
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