New Medicines

Haemophilia A


New molecular entity

Development and Regulatory status

Phase III Clinical Trials
Apr 20Sangamo announces it plans to file a BLA in the US; no further details provided of when, and whether a EU filing will follow [6].
Apr 20Sangamo is responsible for conducting the PF 07055480 PI/II trial and certain manufacturing activities. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialisation activities for PF 07055480 and additional products, if any [5].
Jun 19US FDA has granted regenerative medicine advanced therapy (RMAT) and Fast Track designation for SB-525 gene therapy to treat severe hemophilia A.[5]
May 17Study NCT03061201 now recruiting, estimated primary completion Nov 2021 [3].
May 17Granted orphan drug status in EU & US, and fast track status in US [2].
May 17Sangamo Therapeutics announces an exclusive, global collaboration and license agreement for development and commercialisation of gene therapy programs for haemophilia A, including SB 525, with Pfizer [2].


Single IV infusion of a gene therapy comprising of a recombinant adeno-associated virus (AAV2/6) to deliver a human Factor VIII (hF8) cDNA construct driven by a proprietary synthetic liver specific promoter to the nucleus of liver cells.
It affects 1:4,000 to 1:5,000 live male births worldwide. It is five times as common as haemophilia B [1].
Haemophilia A
Intravenous infusion

Trial or other data

Oct 20Pfizer announces dosing of the first patient in the PIII AFFINE study [9].
Jul 20Pivotal PIII AFFINE study (NCT04370054; C3731003) is due to start recruiting. It will evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in 63 adult male participants with moderately severe or severe hemophilia A (FVIII:C≤1%) for the study duration of 5 years. The study will enroll eligible participants who have completed at least 6 months of routine prophylaxis with FVIII products in the lead-in study C0371004. Study sites not yet stated. Collection of primary outcome data is due to complete Aug 22 [8].
Apr 20The PIII lead-in study (NCT03587116) is recruiting 250 adults in sites including the US, Brazil, Canada, Israel, Japan, EU and UK (trial sites include Newcastle upon Tyne Hospitals NHS Foundation Trust, Non Malignant Haematology Research Unit in Newcastle upon Tyne, Clinical Research Facility in Glasgow, and Department of Haematology in Glasgow). The aim is to establish a minimum of 6 months of prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study. There is no investigational product being administered; subjects will be administering their own standard of care FIX replacement therapy [8].
Apr 20No UK trial sites to date.
Dec 19Pfizer is advancing SB-525 into a PIII registrational clinical study in 2020 and has already commenced enrolling patients into a PIII lead-in study [5].
Dec 19Sangamo and Pfizer announce updated follow-up results from the PI/IIa Alta study. The data showed that SB-525 was generally well tolerated and demonstrated sustained increased Factor VIII (FVIII) levels following treatment with SB-525 through to 44 weeks, the extent of follow-up for the longest treated patient in the 3e13 vg/kg dose cohort. All five patients in the high dose (3e13 vg/kg) cohort rapidly achieved normal levels of Factor VIII, and that Factor VIII levels have been stable and durable in the normal range for the first two patients up to 44 and 37 weeks following treatment respectively, with no bleeding events or factor usage up to a follow up of 44 weeks in the longest treated patient [7].
Jul 19Sangamo and Pfizer announce updated PI/II data showing sustained increase in factor VIII levels. Across the dose cohorts, pts (n=4) demonstrated a dose-dependent increase in FVIII levels and a dose-dependent reduction in the use of FVIII replacement therapy. It was generally well tolerated with 1 reported treatment-related serious adverse event (SAE). This patient experienced hypotension and fever 6 hours after SB-525 infusion; which fully resolved with treatment and discharge was within 24 hours. A 5th pt will be treated soon.[4]
Jun 17PI/II Alta trial to assess safety and tolerability of SB525 in adult patients with severe haemophilia A starts (SB-525-1603; NCT03061201). Primary endpoint will measure the changes in circulating Factor VIII activity, and number of adverse events as assessed by laboratory assessments and vital signs. This open-label trial intends to enrol approximately 20 adult male patients in the US [2].