EMA Grants Marketing Authorization for Sobi and Sanofi's Hemophilia A Treatment, Altuvoct
Innovative Extended Half-Life Technology Enters the European Market
The European Medicines Agency (EMA) has granted marketing authorization for Altuvoct (efanesoctocog alfa), a once-weekly extended half-life FVIII (Factor VIII) replacement therapy for the treatment of Hemophilia A. Altuvoct represents a first-in-class, high-sustained therapy that overcomes the limitations of conventional FVIII treatments by fusing FVIII with von Willebrand factor and an XTEN polypeptide. This EMA approval signifies the official launch of this innovative medicine, which dramatically improves the convenience of dosing compared to existing standard therapies, in 27 European Union (EU) countries. It offers patients an improved quality of life and provides healthcare professionals with a reliable new treatment option.
Overwhelming Efficacy Demonstrated Through XTEND Phase 3 Data
This marketing authorization is based on the results of the XTEND-1 (NCT04161495) study in adults and adolescents, and the XTEND-Kids (NCT04759131) study in children. In the XTEND-1 study, the once-weekly prophylactic Altuvoct regimen reduced the annualized bleeding rate (ABR) by 77% compared to previous FVIII prophylactic regimens (p < 0.001), and maintained FVIII activity levels at or above 40% for approximately 4 days per week. The XTEND-Kids study also demonstrated a median annualized bleeding rate of 0.00 in pediatric patients, exhibiting excellent hemostatic efficacy and a favorable safety profile with no development of FVIII inhibitors. This robust clinical data will be a key driver for future market penetration.
Sobi and Sanofi's Global Partnership Synergy
Altuvoct is the result of a strategic co-development between Sobi (Nasdaq Stockholm: SOBI), a Swedish specialty pharmaceutical company focused on rare diseases, and Sanofi (Nasdaq: SNY), a global biopharmaceutical company. In September 2019, Sobi and Sanofi established a collaborative partnership, with Sobi paying Sanofi an upfront payment of $50 million USD. Under the agreement, Sobi is responsible for the commercialization of Altuvoct in Europe, North Africa, Russia, and the Middle East, while Sanofi is responsible for the distribution and marketing of Altuvoct (marketed as Altuviiio in the United States) in North America and other global regions. With this approval, the financial benefits, including revenue sharing and royalty payments, are expected to be realized.
Anticipated Shift in the European Hemophilia Market, Valued at $3.4 Billion
In 2024, the European hemophilia market is estimated to be approximately $3.41 billion, with Hemophilia A accounting for the largest share. Altuvoct is poised to become a strong competitor in the prophylactic therapy market, which is currently dominated by Roche's blockbuster non-factor therapy, Hemlibra (emicizumab). By maintaining Orphan Drug designation, Altuvoct is expected to secure exclusive market rights in Europe for the next 10 years, providing a solid foundation for maximizing commercial revenue. Sobi plans to initiate discussions with individual countries regarding pricing and reimbursement to begin actual market penetration.
The EMA approval of Altuvoct will create a real and significant competitive dynamic in the European Hemophilia A market, which is estimated at approximately $3.41 billion annually, challenging Roche's Hemlibra, a leading non-factor therapy. The 77% reduction in annualized bleeding rate (ABR) demonstrated in Phase 3 trials (XTEND-1 and XTEND-Kids), along with the maintenance of FVIII activity levels at or above 40% for approximately 3-4 days per week, provides compelling clinical evidence that will likely influence physician prescribing decisions. In the short term, the speed and outcome of pricing and reimbursement negotiations with European health authorities will be a critical factor in determining Altuvoct's initial sales trajectory in Europe. In the long term, the 10-year orphan drug exclusivity will help to prevent competition from biosimilars and other follow-on products, playing a key role in maximizing the long-term pipeline value and cash flow for both Sobi and Sanofi.
Source: EMA (ema)