Fidanacogene elaparvovec is under clinical development by Pfizer and currently in Pre-Registration for Hemophilia B (Factor IX Deficiency). According to GlobalData, Pre-Registration drugs for Hemophilia B (Factor IX Deficiency) have a 100% phase transition success rate (PTSR) indication benchmark for progressing into Marketed. GlobalData’s report assesses how Fidanacogene elaparvovec’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Fidanacogene elaparvovec overview

Fidanacogene Elaparvovec (Beqvez) is an adeno-associated viral (AAV) vector-based gene therapy. It is formulated as solution for intravenous route of administration. Beqvez is indicated for the treatment of adults (aged 18 years or older) with moderately severe to severe hemophilia B (congenital Factor IX deficiency) who are negative for neutralizing antibodies to variant AV serotype Rh74.

Fidanacogene elaparvovec (SPK-9001, PF-06838435) is under development for the treatment of hemophilia B and fabry disease. The therapeutic candidate is administered by intravenous route as a solution. The therapeutic candidate uses an engineered adeno-associated virus to deliver the human FIX gene (AAV8-hFIX19 vector) to liver cells, where FIX is normally made. It is a new molecular entity.

Pfizer overview

Pfizer discovers, develops, manufactures, and commercializes biopharmaceuticals. The company offers products to treat various conditions such as cardiovascular, metabolic and pain, women’s health, cancer, inflammation, immune disorders, and rare diseases. It also provides sterile injectable pharmaceuticals, biosimilars, active pharmaceutical ingredients (APIs) and contract manufacturing services. Pfizer sells its products through wholesalers, retailers, hospitals, individual provider offices, clinics, government agencies and pharmacies. It has major manufacturing facilities in India, China, Japan, Ireland, Italy, Belgium, Germany, Singapore, and the US. The company provides its products in North America, South America, Asia-Pacific, Australia, Europe, Africa, and the Middle East. Pfizer is headquartered in New York, the US.

For a complete picture of Fidanacogene elaparvovec’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 20 February 2024

Premium Insights


The gold standard of business intelligence.

Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.