Valoctocogene roxaparvovec

Despite reporting a Phase 3 win this summer , Pfizer terminated its hemophilia A gene therapy pact with Sangamo Therapeutics in a move that cast a pall on the California biotech’s future. In response to Monday’s news about giroctocogene fitelparvovec, Sangamo’s shares $SGMO tumbled about 50% in Tuesday morning trading. The struggling biotech said earlier this month that it had just enough money to make it to the middle of 2025 after inking a potentially company-saving deal with Astellas . Its financial future is now far less certain. Sangamo had still expected up to $220 million more in regulatory and commercial milestones from Pfizer as part of their tie-up, which began in 2017 with $70 million upfront. Sangamo said Monday it will seek out “all options to commercialize” the gene therapy, including finding a new partner. “While we were surprised and extremely disappointed by Pfizer’s decision to end our collaboration so close to the anticipated BLA and MAA submissions, especially given the compelling pivotal clinical trial data, we appreciate their collaboration in leading a robust and successful clinical development program and for advancing the asset to this important stage,” Sangamo CEO Sandy Macrae said in a statement. Pfizer’s pullback occurs just weeks after it presented more data from the Phase 3 study at the American Society of Hematology’s annual meeting. The New York pharma giant, which got two hemophilia drugs approved this year, attributed the deal’s termination to market factors, an “extensive analysis” of clinical data, feedback from experts and “the slow uptake of hemophilia A gene therapy in patients with moderate-to-severe disease,” a spokesperson said in an email. Earlier this year, Pfizer received FDA approval for Beqvez , its hemophilia B gene therapy, and Hympavzi , a once-weekly medicine for hemophilia A or B. “We believe there is limited interest in an additional gene therapy option in this patient population at this time,” the spokesperson added. The other gene therapy in question is BioMarin’s Roctavian, which has had a patchy uptake since being approved in August 2023 and has since led the drugmaker to reconsider its commercial plans for the gene therapy. At the time of Sangamo and Pfizer’s Phase 3 readout in July, questions circled around whether the duo could make a mark in the field given Roctavian’s precedent. Roche also recently terminated a Phase 3 trial for a hemophilia A gene therapy, though it said it’s working on a new candidate. Meanwhile, Sangamo said it needs more money to finance a Phase 1/2 study next year for ST-503 for idiopathic small fiber neuropathy and to move a prion disease program forward. It also said it’s in “advanced business development discussions” surrounding its Fabry gene therapy program.

Shares in Sangamo lost 50% of their value in after-hours trading following the announcement, which came just before the close on 30 December. Credit: jayk7 via Getty Images. Sangamo Therapeutics’ stock has plummeted by 50% in premarket trading after Pfizer ended the companies’ partnership to co-develop a gene therapy for haemophilia A. Sangamo announced it will regain the development and commercialisation rights to giroctocogene fitelparvovec, an investigational gene therapy for adults with moderately severe to severe haemophilia A. This follows Pfizer’s decision to terminate their global collaboration and license agreement, effective 21 April 2025. Sangamo plans to explore options to advance the programme, including seeking a new collaboration partner. Shares in Sangamo – which had been set to receive up to $220m in milestone payments under the agreement – lost 50% of their value in after-hours trading following the announcement, which came just before the close on 30 December. The decision comes despite positive results from the Phase III AFFINE clinical trial (NCT04370054), which demonstrated that the therapy met its primary goal by reducing the total annualised bleeding rate (ABR) compared to routine Factor VIII (FVIII) replacement prophylaxis. Pfizer previously indicated plans to submit biologics license application (BLA) and marketing authorisation application filings in early 2025 but has now opted not to proceed. Pfizer said in a statement that a slow uptake of haemophilia A gene therapy in patients with moderate to severe disease and “limited interest” in another gene therapy option for the specified patient population prompted its decision. The development of gene therapies for rare diseases is a resource-intensive process that requires advanced technologies, large-scale clinical trials, and navigation of complex regulatory pathways. This has led several pharmaceutical companies to reevaluate or halt gene therapy programmes 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 . While these treatments can potentially provide long-term or curative outcomes, their high development costs and production complexities result in significant price tags. For instance, BioMarin’s US Food and Drug Administration (FDA)-approved haemophilia A gene therapy, Roctavian (valoctocogene roxaparvovec-rvox), launched in 2023 with a $2.9m price point , sparking ongoing debate about the cost-effectiveness of such therapies. Sandy Macrae, CEO of Sangamo Therapeutics, said the therapy has the potential to be life-changing for haemophilia A patients and is well-positioned for regulatory submissions and commercialisation. Macrae expressed disappointment at Pfizer’s decision, particularly given the strong Phase III data, but acknowledged Pfizer’s role in advancing the programme to this stage. Pfizer will continue to monitor trial participants during the transition period. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy