In July 2024, Pfizer confirmed it would end the development of its gene therapy candidate for Duchenne muscular dystrophy (DMD), a devastating and progressive genetic disorder that leads to muscle degeneration. The decision came after the therapy, fordadistrogene movaparvovec, failed to meet its primary and secondary endpoints in a Phase 3 study. The subsequent layoffs primarily impacted a facility in Sanford, North Carolina, which had been a hub for Pfizer's gene therapy research. The DMD program's demise is a stark reminder of the risks involved in drug development, especially in the complex field of gene therapy.
The Failure of the Duchenne Muscular Dystrophy Program
Pfizer's investment in Duchenne muscular dystrophy research began with the acquisition of Bamboo Therapeutics in 2016. The company invested hundreds of millions into its North Carolina facilities to produce gene therapies for DMD and hemophilia. However, the ambitious project came to an abrupt halt when the Phase 3 trial results were revealed in June 2024. The experimental therapy showed no significant improvement in motor function for the young boys with Duchenne who participated in the study. Following this setback, Pfizer announced it would not continue development and subsequently confirmed the elimination of 150 positions directly related to the program at its Sanford facility. An additional 75 positions were cut at the same site a few months later, also tied to the DMD trial's failure.
The Impact of Trial Failure and Restructuring
- Layoffs in North Carolina: The most direct and immediate consequence was the job losses at Pfizer's Sanford and Rocky Mount facilities in North Carolina. This region had become a significant hub for the company's gene therapy and manufacturing operations.
- Strategic Reprioritization: The failure underscored the volatile nature of clinical trials, particularly in advanced therapeutics like gene therapy. It prompted a re-evaluation of Pfizer's rare disease and gene therapy strategy.
- Industry Ripple Effect: The news also rippled through the biopharmaceutical industry, highlighting the challenges of developing and commercializing gene therapies for rare diseases. The company's exit from the DMD race left Sarepta Therapeutics' Elevidys as the only gene therapy option available, though its efficacy has also faced scrutiny.
Withdrawal of Sickle Cell Disease Treatment
Beyond the DMD program, Pfizer made another major move in its rare disease portfolio in 2024. In September, the company voluntarily withdrew its sickle cell disease (SCD) treatment, Oxbryta (voxelotor), from worldwide markets. This decision followed the discovery of a higher risk of complications and deaths among patients in a long-term trial. The drug had received accelerated FDA approval in 2019, but confirmatory trial data led to the reversal. This move marked a significant setback, especially as Oxbryta was a key asset acquired with Global Blood Therapeutics in 2022.
Hemophilia B Gene Therapy Discontinuation
In early 2025, Pfizer also announced the discontinuation of its hemophilia B gene therapy, Beqvez, less than a year after its FDA approval. According to a company spokesperson, the decision was driven by “limited interest” from patients and doctors, reflecting broader concerns within the medical community about the long-term safety, efficacy, and high cost of such one-time treatments. This decision further signaled Pfizer's shift in its gene therapy focus and the competitive challenges within the rare bleeding disorders market.
Additional Rare Disease Portfolio Adjustments
The changes in Pfizer's rare disease strategy are part of a larger cost-cutting campaign announced in late 2023. As part of this realignment, Pfizer also began selling off other early-stage rare disease assets. In 2023, it divested a portfolio of preclinical gene therapies to Alexion, AstraZeneca Rare Disease, and shelved other programs, including candidates for achondroplasia and chronic inflammatory demyelinating polyneuropathy (CIDP). These moves indicate a broader reprioritization of its R&D investments to focus on more promising or synergistic therapeutic areas.
Comparison of Major Rare Disease Program Discontinuation
| Program/Drug | Rare Disease | Reason for Discontinuation | Layoffs Triggered? | Year of Decision |
|---|---|---|---|---|
| Fordadistrogene movaparvovec | Duchenne Muscular Dystrophy | Failed Phase 3 clinical trial | Yes, at Sanford facility | 2024 |
| Oxbryta (voxelotor) | Sickle Cell Disease | Higher risk of complications/deaths in long-term trial | Not directly cited, but part of wider cuts | 2024 |
| Beqvez | Hemophilia B | Limited market interest post-approval | Not directly cited, but part of wider cuts | 2025 |
| Preclinical Programs | Achondroplasia, CIDP, others | Strategic reprioritization and cost-cutting | Not directly cited | 2023 |
The Broader Context of Biopharma Strategy
Pfizer's decisions are part of a trend among large pharmaceutical companies facing immense pressure to deliver consistent growth and return on investment. The high costs, long timelines, and high failure rates of rare disease and gene therapy development make them high-risk ventures. When programs fail or market uptake is slower than expected, companies must make difficult choices to reallocate resources to more promising areas. While devastating for the patients and employees involved, these are often considered necessary strategic moves to ensure the long-term financial health of the company.
For more detailed information on biopharma industry trends and decisions, you can consult articles from Fierce Pharma, which extensively covers the sector.
Conclusion
Pfizer's discontinuation of the Duchenne muscular dystrophy gene therapy and subsequent layoffs were a direct result of a failed Phase 3 clinical trial. This decision, however, was not an isolated incident but part of a larger, multi-year cost realignment and strategic reprioritization effort. The company also withdrew its sickle cell disease drug Oxbryta due to safety concerns and ended its hemophilia B gene therapy due to low market interest, reflecting the immense financial and clinical pressures of developing therapies for rare diseases. For patients and families, each of these decisions represents a lost hope, while for the company, it's a recalibration of resources in a highly competitive and challenging market landscape.
